Anaemias

7 studies

FIH Study of ALN-CFB in patients with PNH and persistent anemia on SOCA RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, FIRST IN HUMAN STUDY OF THE SAFETY, TOLERABILITY, PHARMACOKINETICS, AND PRELIMINARY EFFICACY OF ALN-CFB, AN INVESTIGATIONAL SIRNA THERAPEUTIC AGAINST COMPLEMENT FACTOR B, IN PARTICIPANTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA AND PERSISTENT ANEMIA ON APPROVED C5-INHIBITOR THERAPY

RCT Commercial Anaemias Phase I/II

Population: Adults aged 18 years or older (body weight ≥40 kg) with paroxysmal nocturnal haemoglobinuria (PNH) confirmed by flow cytometry, on a stable dose of an approved C5 inhibitor (eculizumab, ravulizumab, or crovalimab) for at least 24 weeks, with persistent anaemia (haemoglobin ≤10 g/dL) and an adequate reticulocyte count (≥100×10^9/L) despite this therapy.
Intervention: ALN-CFB, an investigational siRNA therapeutic against complement factor B, given in addition to the participant's existing background C5-inhibitor therapy, in this first-in-human study of safety, tolerability, pharmacokinetics, and preliminary efficacy.
Comparator: Placebo, added to background C5-inhibitor therapy, in this randomised, double-blind, placebo-controlled design.
Outcome: Not specified in the NIHR record.

2 recruited (UK total, all sites to date) 2 open sites

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King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Leeds Teaching Hospitals NHS Trust – St James's University Hospital

ClinicalTrials.gov →

TIARAProduction of expanded autologous regulatory T cells to treat patients with aplastic anaemia: A phase I study

Interventional Academic / non-commercial Anaemias Phase I

Population: Adults aged 18 years and older with acquired idiopathic aplastic anaemia (very severe, severe, or non-severe, with no evidence of a constitutional/inherited cause), who lack a matched sibling/haploidentical or matched unrelated donor (or are otherwise ineligible for transplant), are transfusion dependent, and have failed or are ineligible for antithymocyte globulin/ciclosporin (ATG/CSA) and eltrombopag.
Intervention: Expanded autologous regulatory T cells (Tregs), collected by leukapheresis and manufactured under GMP conditions, then administered as cellular therapy using a 3+3 dose-escalation design exploring two dose levels, in this Phase I trial.
Comparator: None (single-arm, non-randomised, open-label Phase I dose-escalation study).
Outcome: The study investigates the safety and tolerability of expanded autologous Tregs in aplastic anaemia, and examines resulting changes in the immune system and whether healthy bone marrow stem cells recover, thereby improving blood counts, after Treg administration.

7 recruited (UK total, all sites to date) 1 open site

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King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)

ClinicalTrials.gov →NIHR public summary →

Alexion PNH Registry M07-001Paroxysmal Noctural Hemoglobinuria (PNH) Registry

Observational Commercial Anaemias

This is an observational registry for patients with paroxysmal nocturnal haemoglobinuria (PNH), sponsored by Alexion. The available NIHR record does not provide a research summary or specific inclusion/exclusion criteria (both listed as 'TBC'), so the study's population, purpose, and data collection details cannot be further characterised from the given text.

23 recruited (UK total, all sites to date) 1 open site

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Cardiff & Vale University LHB – Cardiff & Vale University LHB

ClinicalTrials.gov →

Ascelus-HA multi-centre investigation of use of a digital follow-up pathway utilising the Ascelus app for monitoring long-term blood conditions, incorporating patient, carer, and clinician co-design and evaluation, clinical validation, and safety investigations

Observational Academic / non-commercial Anaemias

Ascelus-H is a non-randomised, multi-arm study evaluating a digital follow-up pathway (the Ascelus app) for monitoring long-term blood conditions, combining co-design, clinical validation, and safety investigation. It comprises: Arm 1, patient/carer co-design via focus groups/interviews on satisfaction and empowerment; Arm 2, healthcare professional co-design and feedback via focus groups/interviews; Arm 3, key opinion leader interviews on implementation, scalability, and commercialisation; Arm 4, a retrospective clinical review of patients on face-to-face, telephone, and virtual follow-up pathways in the 12 months before digital pathway implementation and at 6 and 12 months after, collecting safety and outcome data; and Arm 5, a prospective patient group on the new digital pathway providing outcome, quality-of-life, and empowerment data. Arm 5 participants must be ≥16 years old, have a diagnosis of a relevant stable condition, be under the care of a participating Trust, and be able to access and use the Ascelus platform; those with comorbidities requiring regular clinic review are excluded from Arm 5.

29 recruited (UK total, all sites to date) 2 open sites

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King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Sheffield Teaching Hospitals NHS Foundation Trust – Weston Park Hospital

ISRCTN Registry →NIHR public summary →

COMPLETEA single arm, multicentre observational study to evaluate effectiveness of pegcetacoplan under real world conditions in patients with paroxysmal nocturnal hemoglobinuria (PNH)

Observational Commercial Anaemias

COMPLETE is a 24-month, single-arm, multicentre, observational study evaluating the real-world effectiveness of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria (PNH), a rare acquired chronic blood cell disorder. Approximately 200 patients across 70 sites in Europe, the Middle East, Canada, and Australia will be enrolled and followed prospectively for 24 (±3) months, with retrospective data on prior PNH treatment, transfusions, and healthcare resource use collected for up to 12 months before pegcetacoplan initiation. Eligible patients are ≥18 years with a documented PNH diagnosis who have started or are about to start routine pegcetacoplan treatment; the study collects data on effectiveness, safety, patient- and clinician-reported outcomes, and healthcare resource use as part of routine clinical care, with no protocol-mandated visit schedule.

6 recruited (UK total, all sites to date) 1 open site

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King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)

ClinicalTrials.gov →

INR-CALCollection Of Venous And Capillary Blood For Development And Support Of The LumiraDx INR Assay

Observational Commercial Anaemias

This non-randomised observational study (INR-CAL) collects venous and capillary blood samples from adults over 18 years who have been receiving vitamin K antagonist therapy for at least 4 weeks, to support development and validation of the LumiraDx point-of-care INR assay. Participants currently taking non-vitamin K antagonist oral anticoagulants (e.g., apixaban, dabigatran, edoxaban, rivaroxaban) or with antiphospholipid antibody syndrome are excluded, as are those who took part in the study within the previous 60 days. No further study purpose beyond assay development/support is given in the record.

3,028 recruited (UK total, all sites to date) 2 open sites

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Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Lanarkshire – University Hospital Wishaw

The Molecular Investigation of Unexplained AnaemiasThe Molecular Investigation of Unexplained Anaemias and Related Congenital Anaemias

Observational Academic / non-commercial Open to new sites Anaemias

This non-randomised observational study uses a novel high-throughput sequencing panel (covering 20+ genes) followed by exome/genome sequencing to identify new genes and mutations causing unexplained inherited anaemias, recruiting patients whose diagnostic samples show novel or no identified mutations on the standard panel. Family members are invited for testing to establish whether identified variants segregate with the condition in a pattern consistent with an inherited cause. Eligible participants (or a relative on their behalf) must be able to give informed consent and have persistent anaemia (occurring on more than one occasion), or be a relative of someone under investigation for persistent anaemia; those with evidence of an acquired cause of anaemia (e.g., low B12/folate/ferritin, cytogenetic bone marrow abnormalities) are excluded. The study aims to improve understanding of red blood cell production, with the stated goal of informing new treatments and supporting laboratory-based blood production research.

293 recruited (UK total, all sites to date) 51 open sites

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Alder Hey Children's NHS Foundation Trust – Alder Hey Children's NHS Foundation Trust
Barts Health NHS Trust – The Royal London Hospital
Bristol NHS Foundation Trust – Bristol NHS Foundation Trust
Cardiff & Vale University LHB – Cardiff & Vale University LHB
Chelsea and Westminster Hospital NHS Foundation Trust – Chelsea & Westminster Hospital
Chelsea and Westminster Hospital NHS Foundation Trust – West Middlesex University Hospital
Croydon Health Services NHS Trust – Croydon University Hospital
East Suffolk and North Essex NHS Foundation Trust – Colchester General Hospital
Greater Glasgow and Clyde – Glasgow Royal Infirmary
Greater Glasgow and Clyde – Stobhill Hospital
Hampshire Hospitals NHS Foundation Trust – Basingstoke and North Hampshire Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Leeds Teaching Hospitals NHS Trust – St James's University Hospital
London North West University Healthcare NHS Trust – Ealing Hospital
Manchester University NHS Foundation Trust – Manchester Royal Infirmary
Mid and South Essex NHS Foundation Trust – Southend Hospital
Milton Keynes University Hospital NHS Foundation Trust – Milton Keynes Hospital
Norfolk and Norwich University Hospitals NHS Foundation Trust – Norfolk & Norwich University Hospital
North West Anglia NHS Foundation Trust – Peterborough City Hospital
Northern Care Alliance NHS Foundation Trust – Fairfield General Hospital
Northern Care Alliance NHS Foundation Trust – Rochdale Infirmary
Northern Care Alliance NHS Foundation Trust – Royal Oldham Hospital
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - City Campus
Oxford University Hospitals NHS Foundation Trust – Churchill Hospital
Oxford University Hospitals NHS Foundation Trust – John Radcliffe Hospital
Pennine Acute Hospitals NHS Trust – Rochdale Infirmary
Pennine Acute Hospitals NHS Trust – Royal Oldham Hospital
Royal Devon University Healthcare NHS Foundation Trust – Royal Devon & Exeter Hospital (Wonford)
Royal Free London NHS Foundation Trust – Royal Free Hospital
Royal United Hospitals Bath NHS Foundation Trust – Royal United Hospitals Bath NHS Foundation Trust
Salisbury NHS Foundation Trust – Salisbury District Hospital
Sandwell and West Birmingham Hospitals NHS Trust – City Hospital
Sheffield Children's NHS Foundation Trust – Sheffield Children's Hospital
Somerset NHS Foundation Trust – Musgrove Park Hospital
St George's University Hospitals NHS Foundation Trust – St George's Hospital (Tooting)
Taunton and Somerset NHS Foundation Trust – Musgrove Park Hospital
The Hillingdon Hospitals NHS Foundation Trust – Hillingdon Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary
The Queen Elizabeth Hospital, King's Lynn, NHS Foundation Trust – The Queen Elizabeth Hospital
The Shrewsbury and Telford Hospital NHS Trust – Royal Shrewsbury Hospital
University College London Hospitals NHS Foundation Trust – University College Hospital
University Hospital Southampton NHS Foundation Trust – Southampton General Hospital
University Hospitals Birmingham NHS Foundation Trust – Heartlands Hospital
University Hospitals Plymouth NHS Trust – Derriford Hospital
University Hospitals of North Midlands NHS Trust – Royal Stoke University Hospital
Walsall Healthcare NHS Trust – Manor Hospital
West Hertfordshire Teaching Hospitals NHS Trust – Watford General Hospital
Whittington Health NHS Trust – The Whittington Hospital
Worcestershire Acute Hospitals NHS Trust – Worcestershire Royal Hospital
Wye Valley NHS Trust – Hereford County Hospital
York and Scarborough Teaching Hospitals NHS Foundation Trust – York Hospital

NIHR public summary →

Autoimmune Disorders

9 studies

ARGX-113-2402A Phase 3, Multicenter, Randomized, Double-Blinded, PlaceboControlled, Parallel-Arm Study Followed by an Open-Label Arm to Evaluate the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia

RCT Commercial Autoimmune Disorders Phase III

Population: Adults aged 18 years or older with primary immune thrombocytopenia (ITP) diagnosed for more than 12 months, platelet count below 100x10^9/L, documented insufficient response to at least one prior first-line ITP treatment (e.g. corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs, rituximab, or splenectomy), and a baseline mean platelet count below 30x10^9/L before randomisation.
Intervention: Efgartigimod administered intravenously, followed by an open-label extension arm.
Comparator: Placebo, in the double-blind, placebo-controlled, parallel-arm phase of this randomised Phase 3 study.
Outcome: Not specified in the NIHR record.

4 recruited (UK total, all sites to date) 12 open sites

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Barts Health NHS Trust – The Royal London Hospital
Betsi Cadwaladr University LHB – Ysbyty Glan Clwyd
Bradford Teaching Hospitals NHS Foundation Trust – Bradford Royal Infirmary
Bradford Teaching Hospitals NHS Foundation Trust – NIHR Patient Recruitment Centre - Bradford
East Kent Hospitals University NHS Foundation Trust – Kent & Canterbury Hospital
Greater Glasgow and Clyde – Glasgow Royal Infirmary
Imperial College Healthcare NHS Trust – Hammersmith Hospital
Royal Cornwall Hospitals NHS Trust – Royal Cornwall Hospital (Treliske)
Sheffield Teaching Hospitals NHS Foundation Trust – Royal Hallamshire Hospital
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital
University Hospitals Coventry and Warwickshire NHS Trust – University Hospital (Coventry)
University Hospitals of Leicester NHS Trust – Leicester Royal Infirmary

ClinicalTrials.gov →

Meza ITP StudyA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of Mezagitamab Subcutaneous Injection in Participants with Persistent or Chronic Primary Immune Thrombocytopenia

RCT Commercial Autoimmune Disorders Phase III

Population: Adults aged 18 years or older with primary immune thrombocytopenia (ITP) persisting for at least 3 months, with documented prior treatment response, insufficient response or intolerance to at least one first-line (e.g. corticosteroids) and one second-line (e.g. TPO-RA, rituximab) therapy, and a mean platelet count below 30,000/µL.
Intervention: Mezagitamab administered by subcutaneous injection.
Comparator: Placebo, in this randomised, double-blind, placebo-controlled Phase 3 study.
Outcome: Not specified in the NIHR record.

14 recruited (UK total, all sites to date) 25 open sites

View open sites (25)

Barts Health NHS Trust – The Royal London Hospital
Bristol NHS Foundation Trust – Bristol Royal Infirmary
Cardiff & Vale University LHB – University Hospital of Wales
East Suffolk and North Essex NHS Foundation Trust – Ipswich Hospital
Grampian – Aberdeen Royal Infirmary
Greater Glasgow and Clyde – Glasgow Royal Infirmary
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
Leeds Teaching Hospitals NHS Trust – St James's University Hospital
Liverpool University Hospitals NHS Foundation Trust – Royal Liverpool University Hospital
North Cheshire and Mersey NHS Foundation Trust – Warrington Hospital
North Cumbria Integrated Care NHS Foundation Trust – Cumberland Infirmary
Northumbria Healthcare NHS Foundation Trust – North Tyneside General Hospital
Oxford University Hospitals NHS Foundation Trust – Churchill Hospital
Royal Cornwall Hospitals NHS Trust – Royal Cornwall Hospital (Treliske)
Royal Free London NHS Foundation Trust – Royal Free Hospital
South Tyneside and Sunderland NHS Foundation Trust – Sunderland Royal Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary
United Lincolnshire Teaching Hospitals NHS Trust – Lincoln County Hospital
University College London Hospitals NHS Foundation Trust – University College Hospital
University Hospitals Dorset NHS Foundation Trust – Poole Hospital
University Hospitals Dorset NHS Foundation Trust – Royal Bournemouth Hospital
University Hospitals Plymouth NHS Trust – Derriford Hospital
University Hospitals of Leicester NHS Trust – Leicester Royal Infirmary
University Hospitals of North Midlands NHS Trust – Royal Stoke University Hospital

ClinicalTrials.gov →

Ph 3, Mavorixafor in Participants w/ Congenital or Acquired Primary Autoimmune or Idiopathic CNDA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Participants with Congenital or Acquired Primary Autoimmune or Idiopathic Chronic Neutropenic Disorders

RCT Commercial Autoimmune Disorders Phase III

Population: Participants ≥12 years of age with congenital or acquired primary autoimmune/idiopathic chronic neutropenic disorders (CN) diagnosed ≥6 months prior to screening, confirmed trough absolute neutrophil count <1500 cells/µL, a history of recurrent/serious infections in the preceding 12 months, and body weight ≥15 kg.
Intervention: Mavorixafor plus background therapy (e.g. G-CSF, immunoglobulin replacement, prophylactic antibiotics, or watchful waiting), dosed orally once daily: adults and adolescents >50 kg receive 400 mg, adolescents ≤50 kg receive 200 mg.
Comparator: Placebo plus background therapy, randomised 1:1, stratified by CN type and G-CSF use at screening.
Outcome: Not specified in the NIHR record

11 recruited (UK total, all sites to date) 13 open sites

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Bristol NHS Foundation Trust – Bristol Haematology & Oncology Centre
Cambridge University Hospitals NHS Foundation Trust – Addenbrooke's Hospital
Cardiff & Vale University LHB – Noahs Ark Childrens Hospital for Wales
Cardiff & Vale University LHB – University Hospital of Wales
Great Ormond Street Hospital for Children NHS Foundation Trust – Great Ormond Street Hospital Central London Site
Imperial College Healthcare NHS Trust – Hammersmith Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Milton Keynes University Hospital NHS Foundation Trust – Milton Keynes Hospital
Oxford University Hospitals NHS Foundation Trust – Nuffield Orthopaedic Centre
Royal Free London NHS Foundation Trust – Barnet Hospital
Royal Free London NHS Foundation Trust – Royal Free Hospital
St George's University Hospitals NHS Foundation Trust – St George's Hospital (Tooting)
University Hospitals Plymouth NHS Trust – Derriford Hospital

ClinicalTrials.gov →

Study of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell DisordersA Phase 2a, Open-Label, Multicenter Study of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders

RCT Commercial Autoimmune Disorders Phase II

Population: Adults (≥18 years) with primary immune thrombocytopenia (ITP) or warm autoimmune haemolytic anaemia (wAIHA) that is persistent/chronic and active with an indication for treatment, no history of splenectomy, and a documented transient response to at least one prior early-line therapy (e.g., corticosteroids, IVIG, rituximab), including ≥1 standard course of rituximab given ≥6 months earlier.
Intervention: Tafasitamab (an anti-CD19 antibody, inferred from the exclusion of prior anti-CD19 therapy) given in this Phase 2a, open-label, multicentre study.
Comparator: None specified — the title describes an open-label design with no placebo or active comparator arm mentioned in the available text; the record's randomisation field is marked 'Randomised' but no control arm is described.
Outcome: Not specified in the NIHR record.

2 recruited (UK total, all sites to date) 4 open sites

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Barts Health NHS Trust – St Bartholomew's Hospital
Barts Health NHS Trust – The Royal London Hospital
Hull University Teaching Hospitals NHS Trust – Castle Hill Hospital
University Hospitals Plymouth NHS Trust – Derriford Hospital

ClinicalTrials.gov →

Study to Investigate the Safety and Efficacy of Pirtobrutinib in Adults with Immune ThrombocytopeniaA Phase 1/2, Dose-finding Study Investigating the Safety and Efficacy of Pirtobrutinib in Adults with Immune Thrombocytopenia

RCT Commercial Autoimmune Disorders Phase I/II

Population: Adults ≥18 with primary immune thrombocytopenia (ITP) of at least 3 months' duration, with a documented platelet-count response (≥2 counts ≥50,000/µL) to at least one prior line of therapy (including splenectomy), now relapsed or treatment-resistant with no available beneficial therapies, and a platelet count <30,000/µL on 2 occasions ≥5 days apart within 15 days of enrolment.
Intervention: Pirtobrutinib, given as part of a Phase 1/2 dose-finding design (specific doses not detailed in the available record).
Comparator: Not specified in the NIHR record — the study is randomised, but no placebo or open-label wording is given in the title/summary.
Outcome: Not specified in the NIHR record

1 recruited (UK total, all sites to date) 10 open sites

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Barts Health NHS Trust – The Royal London Hospital
Bristol NHS Foundation Trust – Bristol Haematology & Oncology Centre
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Leeds Teaching Hospitals NHS Trust – St James's University Hospital
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - Queen's Medical Centre Campus
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - Ropewalk House
University College London Hospitals NHS Foundation Trust – University College Hospital
University Hospitals of Leicester NHS Trust – Leicester Royal Infirmary

ClinicalTrials.gov →

APL-3007Safety and Tolerability of APL-3007 Administered as a Single Dose in Addition to Background Therapy with a C5 Inhibitor in Adults with Paroxysmal Nocturnal Hemoglobinuria

RCT Commercial Autoimmune Disorders Phase I

Population: Adults aged 18 years or older with PNH, treated with a stable dose of ravulizumab for at least the previous 6 months, with haemoglobin below 10.5 g/dL despite therapy, and adequate reticulocyte count (>1x ULN), platelet count (>100x10^9/L), and absolute neutrophil count (>500/mm3).
Intervention: A single dose of APL-3007, an investigational agent, administered in addition to background ravulizumab therapy in adults with PNH.
Comparator: Not explicitly stated in the available record; the study is randomised, with participants enrolled across dose cohorts 1 to 4, but no placebo or blinding status is specified in the title or given text.
Outcome: Not specified in the NIHR record.

1 recruited (UK total, all sites to date) 2 open sites

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King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Leeds Teaching Hospitals NHS Trust – St James's University Hospital

ISRCTN Registry →

GUMBITA pilot study to investigate the gut microbiome in patients with immune thrombocytopenia (ITP)

Observational Academic / non-commercial Autoimmune Disorders

This pilot observational study investigates whether the gut microbiome differs in patients with immune thrombocytopenia (ITP) compared with healthy controls, building on evidence implicating infections such as H. pylori and gut microbiome disturbance in ITP pathophysiology. It aims to recruit 60 participants across three groups: 20 with moderate-to-severe recurrent ITP, 20 with refractory ITP, and 20 healthy controls (preferentially relatives of enrolled ITP patients); eligible ITP patients are adults aged 18 or over who have had a platelet count <=50 x10^9/L at some point during their disease. Participants provide blood, urine and stool samples and complete four questionnaires at a single clinic visit, with no follow-up required. The aim is to identify gut microbiome-related biomarkers and clues for treatment that could inform a future prospective trial modifying the gut microbiome (e.g. via diet, antibiotics, or faecal microbiota transplant) in ITP.

37 recruited (UK total, all sites to date) 0 open sites

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No open sites currently listed.

NIHR public summary →

UK TTP RegistryUnited Kingdom Thrombotic Thrombocytopenic Purpura Registry: A prospective study of Thrombotic Thrombocytopenic Purpura in the UK

Observational Academic / non-commercial Open to new sites Autoimmune Disorders

This is a prospective UK registry of patients with a clinical diagnosis of acute thrombotic thrombocytopenic purpura (TTP), defined by thrombocytopenia and microangiopathic haemolytic anaemia (MAHA), which may be associated with clinical evidence of organ compromise. There is no age restriction for entry; patients are added to the registry following consent. Patients with thrombocytopenia or MAHA attributable to other causes (e.g. Haemolytic Uraemic Syndrome, Disseminated Intravascular Coagulation, pre-eclampsia/HELLP syndrome, or antiphospholipid syndrome) or with cancer- or transplant-associated MAHA are excluded. No further study purpose or objective beyond prospective registry data collection is stated in the available text.

1,582 recruited (UK total, all sites to date) 40 open sites

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Birmingham Women's and Children's NHS Foundation Trust – Birmingham Children's Hospital
Bristol NHS Foundation Trust – Bristol Haematology & Oncology Centre
Cambridge University Hospitals NHS Foundation Trust – Addenbrooke's Hospital
Cardiff & Vale University LHB – Cardiff & Vale University LHB
Chelsea and Westminster Hospital NHS Foundation Trust – Chelsea & Westminster Hospital
Epsom and St Helier University Hospitals NHS Trust – Epsom Hospital
Great Ormond Street Hospital for Children NHS Foundation Trust – Great Ormond Street Hospital Central London Site
Greater Glasgow and Clyde – Beatson West of Scotland Cancer Centre
Greater Glasgow and Clyde – Gartnavel General Hospital
Greater Glasgow and Clyde – Glasgow Royal Infirmary
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
King's College Hospital NHS Foundation Trust – King's College Hospital NHS Foundation Trust
Leeds Teaching Hospitals NHS Trust – Leeds Teaching Hospitals NHS Trust
Liverpool University Hospitals NHS Foundation Trust – Royal Liverpool University Hospital
Lothian – Royal Infirmary of Edinburgh at Little France
Mid Yorkshire Teaching NHS Trust – Pinderfields General Hospital
Norfolk and Norwich University Hospitals NHS Foundation Trust – Norfolk & Norwich University Hospital
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - City Campus
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - Queen's Medical Centre Campus
Oxford University Hospitals NHS Foundation Trust – Churchill Hospital
Poole Hospital NHS Foundation Trust – Poole General Hospital
Portsmouth Hospitals University NHS Trust – Queen Alexandra Hospital
Royal Cornwall Hospitals NHS Trust – Royal Cornwall Hospital (Treliske)
Royal Devon University Healthcare NHS Foundation Trust – Royal Devon & Exeter Hospital (Wonford)
Royal Liverpool and Broadgreen University Hospitals NHS Trust – The Royal Liverpool University Hospital
Sandwell and West Birmingham Hospitals NHS Trust – City Hospital
Sheffield Teaching Hospitals NHS Foundation Trust – Royal Hallamshire Hospital
St George's University Hospitals NHS Foundation Trust – St George's Hospital (Tooting)
The Queen Elizabeth Hospital, King's Lynn, NHS Foundation Trust – The Queen Elizabeth Hospital
University College London Hospitals NHS Foundation Trust – University College Hospital
University Hospital Southampton NHS Foundation Trust – Southampton General Hospital
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital
University Hospitals Coventry and Warwickshire NHS Trust – Coventry City Centre Health Facility
University Hospitals Coventry and Warwickshire NHS Trust – University Hospital (Coventry)
University Hospitals Dorset NHS Foundation Trust – Poole Hospital
University Hospitals Plymouth NHS Trust – Derriford Hospital
University Hospitals Plymouth NHS Trust – University Hospitals Plymouth NHS Trust
University Hospitals of Leicester NHS Trust – Leicester Royal Infirmary

NIHR public summary →

UKAITPRUnited Kingdom Adult Idiopathic Thrombocytopenic Purpura (ITP) Registry: An Investigation of Disease Progression, Treatment Effectiveness, and Co-morbid Conditions

Observational Academic / non-commercial Open to new sites Autoimmune Disorders

The UK Adult ITP Registry (UKAITPR) is a patient registry that collects clinical data (co-morbid conditions, ITP-specific treatments, laboratory results, and bleeding events) and biological samples (whole blood or saliva) from adult patients with primary immune thrombocytopenia (ITP), diagnosed according to the 2010 International Consensus Report criteria. Its purpose is to investigate outstanding questions regarding disease progression, treatment effectiveness, and co-morbid burden in ITP. Eligible participants are adults aged 18 years or older with a diagnosis of primary ITP; no exclusion criteria are specified.

6,209 recruited (UK total, all sites to date) 148 open sites

View open sites (148)

Airedale NHS Foundation Trust – Airedale General Hospital
Aneurin Bevan University LHB – Aneurin Bevan University LHB
Aneurin Bevan University LHB – Nevill Hall Hospital
Aneurin Bevan University LHB – Royal Gwent Hospital
Barking, Havering and Redbridge University Hospitals NHS Trust – Queen's Hospital
Barts Health NHS Trust – Newham General Hospital
Barts Health NHS Trust – The Royal London Hospital
Barts and The London NHS Trust – The Royal London Hospital
Basildon and Thurrock University Hospitals NHS Foundation Trust – Basildon University Hospital
Bedford Hospital NHS Trust – Bedford Hospital
Betsi Cadwaladr University LHB – Wrexham Maelor Hospital
Birmingham Women's and Children's NHS Foundation Trust – Birmingham Women's Hospital
Bolton NHS Foundation Trust – Royal Bolton Hospital
Bradford Teaching Hospitals NHS Foundation Trust – Bradford Royal Infirmary
Brighton and Sussex University Hospitals NHS Trust – Royal Sussex County Hospital
Bristol NHS Foundation Trust – Bristol Haematology & Oncology Centre
Calderdale and Huddersfield NHS Foundation Trust – Calderdale Royal Hospital
Cambridge University Hospitals NHS Foundation Trust – Addenbrooke's Hospital
Cardiff & Vale University LHB – Cardiff & Vale University LHB
Chelsea and Westminster Hospital NHS Foundation Trust – West Middlesex University Hospital
Chesterfield Royal Hospital NHS Foundation Trust – Chesterfield Royal Hospital
City Hospitals Sunderland NHS Foundation Trust – Sunderland Royal Hospital
Countess of Chester Hospital NHS Foundation Trust – Countess of Chester Hospital
County Durham and Darlington NHS Foundation Trust – Darlington Memorial Hospital
Cwm Taf Morgannwg University Local Health Board – Prince Charles Hospital Site
Dartford and Gravesham NHS Trust – Darent Valley Hospital
Doncaster and Bassetlaw Teaching Hospitals NHS Foundation Trust – Doncaster Royal Infirmary
Dorset County Hospital NHS Foundation Trust – Dorset County Hospital
East Kent Hospitals University NHS Foundation Trust – Kent & Canterbury Hospital
East Suffolk and North Essex NHS Foundation Trust – Colchester General Hospital
East Sussex Healthcare NHS Trust – East Sussex Healthcare NHS Trust
East Sussex Healthcare NHS Trust – Eastbourne District General Hospital
Epsom and St Helier University Hospitals NHS Trust – Epsom Hospital
Epsom and St Helier University Hospitals NHS Trust – St Helier Hospital
Forth Valley – Falkirk Community Hospital
Forth Valley – Forth Valley Royal Hospital
Frimley Health NHS Foundation Trust – Wexham Park Hospital
Gateshead Health NHS Foundation Trust – Queen Elizabeth Hospital
Gloucestershire Hospitals NHS Foundation Trust – Gloucestershire Royal Hospital
Great Western Hospitals NHS Foundation Trust – The Great Western Hospital
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Hampshire Hospitals NHS Foundation Trust – Basingstoke and North Hampshire Hospital
Harrogate and District NHS Foundation Trust – Harrogate District Hospital
Hull University Teaching Hospitals NHS Trust – Castle Hill Hospital
Hywel Dda University LHB – Bronglais General Hospital
Hywel Dda University LHB – Glangwili General Hospital
Hywel Dda University LHB – Glangwili Hospital Child Health Section
Hywel Dda University LHB – Prince Philip Hospital
Hywel Dda University LHB – Withybush General Hospital
Isle of Wight NHS Trust – St Mary's Hospital
James Paget University Hospitals NHS Foundation Trust – James Paget University Hospital
Kettering General Hospital NHS Foundation Trust – Kettering General Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
King's College Hospital NHS Foundation Trust – Princess Royal University Hospital
Kingston and Richmond NHS Foundation Trust – Kingston Hospital
Lanarkshire – University Hospital Hairmyres
Lanarkshire – University Hospital Monklands
Lanarkshire – University Hospital Wishaw
Leeds Teaching Hospitals NHS Trust – St James's University Hospital
Lewisham and Greenwich NHS Trust – Queen Elizabeth Hospital
Lewisham and Greenwich NHS Trust – University Hospital Lewisham
Liverpool University Hospitals NHS Foundation Trust – Royal Liverpool University Hospital
London North West University Healthcare NHS Trust – Ealing Hospital
London North West University Healthcare NHS Trust – Northwick Park Hospital
Lothian – Royal Infirmary of Edinburgh at Little France
Manchester University NHS Foundation Trust – Manchester Royal Infirmary
Manchester University NHS Foundation Trust – Wythenshawe Hospital
Medway NHS Foundation Trust – Medway Maritime Hospital
Medway NHS Foundation Trust – Medway NHS Foundation Trust
Mid Yorkshire Teaching NHS Trust – Pinderfields General Hospital
Mid and South Essex NHS Foundation Trust – Basildon Hospital
Mid and South Essex NHS Foundation Trust – Southend Hospital
Milton Keynes University Hospital NHS Foundation Trust – Milton Keynes Hospital
NHS North East and North Cumbria ICB - 00N – South Tyneside District Hospital
Norfolk and Norwich University Hospitals NHS Foundation Trust – Norfolk & Norwich University Hospital
North Bristol NHS Trust – Southmead Hospital
North Cumbria Integrated Care NHS Foundation Trust – Cumberland Infirmary
North Cumbria Integrated Care NHS Foundation Trust – North Cumbria Integrated Care NHS Foundation Trust
North Middlesex University Hospital NHS Trust – North Middlesex Hospital
North West Anglia NHS Foundation Trust – Hinchingbrooke Hospital
North West Anglia NHS Foundation Trust – Peterborough City Hospital
Northampton General Hospital NHS Trust – Northampton General Hospital (Acute)
Northern Care Alliance NHS Foundation Trust – Royal Oldham Hospital
Northumbria Healthcare NHS Foundation Trust – North Tyneside General Hospital
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - City Campus
Oxford University Hospitals NHS Foundation Trust – Churchill Hospital
Portsmouth Hospitals University NHS Trust – Queen Alexandra Hospital
Royal Berkshire NHS Foundation Trust – Royal Berkshire Hospital
Royal Cornwall Hospitals NHS Trust – Royal Cornwall Hospital (Treliske)
Royal Devon University Healthcare NHS Foundation Trust – Royal Devon & Exeter Hospital (Wonford)
Royal Free London NHS Foundation Trust – Barnet Hospital
Royal Free London NHS Foundation Trust – Chase Farm Hospital
Royal Free London NHS Foundation Trust – North Middlesex Hospital
Royal Liverpool and Broadgreen University Hospitals NHS Trust – The Royal Liverpool University Hospital
Royal Surrey County Hospital NHS Foundation Trust – Royal Surrey County Hospital
Royal United Hospitals Bath NHS Foundation Trust – Royal United Hospital
Salisbury NHS Foundation Trust – Salisbury District Hospital
Sandwell and West Birmingham Hospitals NHS Trust – City Hospital
Sandwell and West Birmingham Hospitals NHS Trust – Midland Metropolitan University Hospital
Sandwell and West Birmingham Hospitals NHS Trust – Sandwell General Hospital
Sheffield Teaching Hospitals NHS Foundation Trust – Royal Hallamshire Hospital
Sherwood Forest Hospitals NHS Foundation Trust – King's Mill Hospital
Somerset NHS Foundation Trust – Musgrove Park Hospital
South Tees Hospitals NHS Foundation Trust – The James Cook University Hospital
South Tyneside NHS Foundation Trust – South Tyneside District Hospital
South Tyneside and Sunderland NHS Foundation Trust – South Tyneside District Hospital
South Tyneside and Sunderland NHS Foundation Trust – Sunderland Royal Hospital
South Warwickshire University NHS Foundation Trust – Warwick Hospital
Southport and Ormskirk Hospital NHS Trust – Southport General Infirmary
St George's University Hospitals NHS Foundation Trust – St George's Hospital (Tooting)
Surrey and Sussex Healthcare NHS Trust – East Surrey Hospital
Taunton and Somerset NHS Foundation Trust – Musgrove Park Hospital
The Dudley Group NHS Foundation Trust – Russells Hall Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary
The Queen Elizabeth Hospital, King's Lynn, NHS Foundation Trust – The Queen Elizabeth Hospital
The Rotherham NHS Foundation Trust – Rotherham District General Hospital
The Royal Bournemouth and Christchurch Hospitals NHS Foundation Trust – Royal Bournemouth General Hospital
The Royal Wolverhampton NHS Trust – New Cross Hospital
The Shrewsbury and Telford Hospital NHS Trust – Royal Shrewsbury Hospital
The Shrewsbury and Telford Hospital NHS Trust – The Shrewsbury and Telford Hospital NHS Trust
The Walton Centre NHS Foundation Trust – Ysbyty Gwynedd Hospital (YG NHS Trust)
Torbay and South Devon NHS Foundation Trust – Torbay Hospital
United Lincolnshire Teaching Hospitals NHS Trust – Grantham & District Hospital
United Lincolnshire Teaching Hospitals NHS Trust – Lincoln County Hospital
United Lincolnshire Teaching Hospitals NHS Trust – Pilgrim Hospital
University College London Hospitals NHS Foundation Trust – University College Hospital
University Hospital Southampton NHS Foundation Trust – Southampton General Hospital
University Hospital Southampton NHS Foundation Trust – University Hospital Southampton NHS Foundation Trust
University Hospitals Birmingham NHS Foundation Trust – Good Hope Hospital
University Hospitals Birmingham NHS Foundation Trust – Heartlands Hospital
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital
University Hospitals Coventry and Warwickshire NHS Trust – University Hospital (Coventry)
University Hospitals Dorset NHS Foundation Trust – Poole Hospital
University Hospitals Dorset NHS Foundation Trust – Royal Bournemouth Hospital
University Hospitals Plymouth NHS Trust – Derriford Hospital
University Hospitals Sussex NHS Foundation Trust – Royal Sussex County Hospital
University Hospitals Sussex NHS Foundation Trust – St Richard's Hospital
University Hospitals Sussex NHS Foundation Trust – Worthing Hospital
University Hospitals of Derby and Burton NHS Foundation Trust – Burton Hospital
University Hospitals of Derby and Burton NHS Foundation Trust – Royal Derby Hospital
University Hospitals of Leicester NHS Trust – Leicester Royal Infirmary
West Hertfordshire Teaching Hospitals NHS Trust – Watford General Hospital
West Suffolk NHS Foundation Trust – West Suffolk Hospital
Whittington Health NHS Trust – The Whittington Hospital
Worcestershire Acute Hospitals NHS Trust – Worcestershire Royal Hospital
Wye Valley NHS Trust – Hereford County Hospital
York and Scarborough Teaching Hospitals NHS Foundation Trust – Scarborough General Hospital
York and Scarborough Teaching Hospitals NHS Foundation Trust – York Hospital

NIHR public summary →

Bleeding Disorders

16 studies

Type III VWD (IS)A PHASE III, MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF EMICIZUMAB PROPHYLAXIS IN PATIENTS WITH TYPE 3 VON WILLEBRAND DISEASE

RCT Commercial Bleeding Disorders Phase III

Population: Patients aged 2 years and older with a confirmed diagnosis of Type 3 von Willebrand disease (VWF antigen and VWF activity both <5 IU/dL, and FVIII level <=10 IU/dL from a single blood draw), with documented prior use of either on-demand haemostatic therapy or scheduled prophylaxis as standard of care.
Intervention: Emicizumab prophylaxis, delivered in this Phase 3, multicentre, open-label, randomised study across arms including Arm A (emicizumab following previous standard-of-care on-demand therapy) and Arm C (emicizumab following previous standard-of-care prophylaxis in a preceding study); specific emicizumab dose/schedule is not given in the provided text.
Comparator: Standard-of-care on-demand treatment, continued unchanged in Arm B.
Outcome: Not specified in the NIHR record.

2 recruited (UK total, all sites to date) 5 open sites

View open sites (5)

Great Ormond Street Hospital for Children NHS Foundation Trust – Great Ormond Street Hospital Central London Site
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Manchester University NHS Foundation Trust – Manchester Royal Infirmary
Manchester University NHS Foundation Trust – Royal Manchester Children's Hospital
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital

ClinicalTrials.gov →

HMB-001-201A Clinical Study to Assess the Safety and Efficacy of Sutacimig in Participants with Congenital Factor VII Deficiency

RCT Commercial Bleeding Disorders Phase II

Population: Adults aged 18-60 with congenital Factor VII deficiency (Factor VII:C activity <10% on at least two measurements) and a severe bleeding history (major bleeding event and/or prior treatment with recombinant FVIIa or fresh frozen plasma), without active major bleeding at screening.
Intervention: Administration of sutacimig in this Phase II randomised study assessing safety and efficacy in congenital FVII deficiency.
Comparator: Not specified in the NIHR record — the study is randomised, but the comparator arm (e.g. placebo, alternative dose, or standard care) is not described in the available text.
Outcome: Not specified in the NIHR record.

2 recruited (UK total, all sites to date) 2 open sites

View open sites (2)

Barts Health NHS Trust – Barts Health NHS Trust
Barts Health NHS Trust – The Royal London Hospital

ClinicalTrials.gov →

Safety, tolerability, PK, PD, and efficacy of HMB-002 in participants with Von Willebrand DiseaseA Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-002 in Participants with Von Willebrand Disease

RCT Commercial Bleeding Disorders Phase I/II

Population: Adults aged 18 to under 65 years, weighing 60-110 kg, with congenital Type 1 Von Willebrand Disease (residual VWF activity <=40 IU/dL, FVIII:C <=70 IU/dL); Part B participants must additionally have symptomatic bleeding events recorded during participation in a prior observational study (HMB-002-101_SCR).
Intervention: Administration of HMB-002 in this Phase 1/2 randomised study assessing safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy.
Comparator: Not specified in the NIHR record — the study is randomised, but the comparator arm is not described in the available text.
Outcome: Not specified in the NIHR record.

1 recruited (UK total, all sites to date) 14 open sites

View open sites (14)

Barts Health NHS Trust – The Royal London Hospital
Bristol NHS Foundation Trust – Bristol Royal Infirmary
Cardiff & Vale University LHB – University Hospital of Wales
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Hampshire Hospitals NHS Foundation Trust – Basingstoke and North Hampshire Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
Leeds Teaching Hospitals NHS Trust – Leeds Teaching Hospitals NHS Trust
Leeds Teaching Hospitals NHS Trust – St James's University Hospital
Liverpool University Hospitals NHS Foundation Trust – Royal Liverpool University Hospital
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - Queen's Medical Centre Campus
Sheffield Teaching Hospitals NHS Foundation Trust – Royal Hallamshire Hospital
St George's University Hospitals NHS Foundation Trust – St George's Hospital (Tooting)
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital

ClinicalTrials.gov →

VOYAGER 2Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Peroral Doses of Inno8 in People with Haemophilia A

RCT Commercial Bleeding Disorders Phase I

Population: Male participants aged 18–64 years with congenital haemophilia A (FVIII activity ≤15%), with or without FVIII inhibitors.
Intervention: Multiple ascending peroral (oral) doses of Inno8, assessed for safety, tolerability, pharmacokinetics and pharmacodynamics in a randomised, dose-escalation design.
Comparator: Not specified in the NIHR record — the study is randomised across ascending oral dose levels of Inno8, but no placebo or open-label wording is given in the available text.
Outcome: Not specified in the NIHR record

3 recruited (UK total, all sites to date) 5 open sites

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Guy's and St Thomas' NHS Foundation Trust – St Thomas' Hospital
Oxford University Hospitals NHS Foundation Trust – Churchill Hospital
Royal Free London NHS Foundation Trust – Royal Free Hospital
Sheffield Teaching Hospitals NHS Foundation Trust – Royal Hallamshire Hospital
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital

ClinicalTrials.gov →

B7841007 - HaemA OLEAN OPEN-LABEL EXTENSION STUDY TO EVALUATE THE LONG-TERM SAFETY, TOLERABILITY, AND EFFICACY OF MARSTACIMAB PROPHYLAXIS IN SEVERE (COAGULATION FACTOR ACTIVITY <1%) HEMOPHILIA A PARTICIPANTS WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY ≤2%) WITH OR WITHOUT INHIBITORS

Interventional Commercial Bleeding Disorders Phase III

Population: Adolescent and adult participants (12 to <75 years) with severe haemophilia A (FVIII activity <1%) with or without inhibitors, or moderately severe to severe haemophilia B (FIX activity <=2%) with or without inhibitors, and paediatric participants (from age 1 year) with the same disease definitions, all of whom successfully completed (without early termination) the parent studies B7841005 or B7841008.
Intervention: Long-term open-label extension treatment with once-weekly subcutaneous marstacimab prophylaxis, continued at the dose established during the participant's parent study.
Comparator: None (single-arm, open-label, non-randomised extension study).
Outcome: Not specified in the NIHR record.

2 recruited (UK total, all sites to date) 5 open sites

View open sites (5)

Birmingham Women's and Children's NHS Foundation Trust – Birmingham Children's Hospital
Birmingham Women's and Children's NHS Foundation Trust – Birmingham Women's Hospital
Guy's and St Thomas' NHS Foundation Trust – St Thomas' Hospital
Manchester University NHS Foundation Trust – Royal Manchester Children's Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary

ClinicalTrials.gov →

Paediatric Haemophilia A&BAN OPEN-LABEL STUDY IN PEDIATRIC (<18 YEARS OF AGE), SEVERE HEMOPHILIA A PARTICIPANTS (COAGULATION FACTOR ACTIVITY <1%) WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY ≤2%) WITH OR WITHOUT INHIBITORS COMPARING 12 MONTHS OF HISTORICAL STANDARD TREATMENT TO MARSTACIMAB PROPHYLAXIS

Interventional Commercial Bleeding Disorders Phase III

Population: Male paediatric participants (<18 years, across three age/weight bands: 12-<18y ≥35kg, 6-<12y ≥19kg, 1-<6y) with severe haemophilia A (FVIII activity <1%) or moderately severe to severe haemophilia B (FIX activity ≤2%), with or without inhibitors, who have ≥1 year of documented bleed/treatment diary or medical-record data on FVIII/FIX replacement or bypass agent use.
Intervention: Marstacimab prophylaxis given in an open-label design.
Comparator: 12 months of historical standard treatment (the participant's own prior factor replacement/bypass agent regimen), per the title "comparing 12 months of historical standard treatment to marstacimab prophylaxis" — a historical rather than concurrent randomised comparator, consistent with the record's non-randomised status.
Outcome: Not specified in the NIHR record.

3 recruited (UK total, all sites to date) 8 open sites

View open sites (8)

Birmingham Women's and Children's NHS Foundation Trust – Birmingham Children's Hospital
Cardiff & Vale University LHB – University Hospital of Wales
Epsom and St Helier University Hospitals NHS Trust – St Helier Hospital
Guy's and St Thomas' NHS Foundation Trust – St Thomas' Hospital
Leeds Teaching Hospitals NHS Trust – Clarendon Wing, Leeds General Infirmary
Leeds Teaching Hospitals NHS Trust – Leeds General Infirmary
Manchester University NHS Foundation Trust – Royal Manchester Children's Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary

ClinicalTrials.gov →

VIVID-6A Prospective, Multicenter, Open-label, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of Prophylactic VGA039 in Adolescent and Adult Patients with von Willebrand Disease

Interventional Commercial Bleeding Disorders Phase III

Population: Adult and adolescent patients aged 18–75 years with a documented diagnosis of von Willebrand disease of any type and a historical annualised bleed rate ≥12 (excluding menstrual and subcutaneous bleeds) over the 6 months prior to screening, judged suitable candidates for routine prophylaxis.
Intervention: Prophylactic administration of VGA039 in an open-label, non-randomised design (specific dosing regimen not detailed in the available record).
Comparator: None (single-arm, open-label study; non-randomised).
Outcome: Not specified in the NIHR record

2 recruited (UK total, all sites to date) 5 open sites

View open sites (5)

Barts Health NHS Trust – The Royal London Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
Royal Free London NHS Foundation Trust – Royal Free Hospital
University Hospital Southampton NHS Foundation Trust – Southampton General Hospital
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital

ClinicalTrials.gov →

BEYOND-9A Two-Part Open-Label Study of REGV131-LNP1265, a CRISPR/Cas9-Based Coagulation Factor IX Gene Insertion Therapy in Participants with Hemophilia B

Interventional Commercial Bleeding Disorders Phase I/II

Population: Males over 18 years of age with confirmed severe or moderately severe haemophilia B (FIX functional activity <=2%, or a documented genotype known to produce severe disease), currently on FIX prophylaxis with at least 150 prior exposure days to a FIX product, who have completed a lead-in period of at least 6 months of bleed-rate (ABR) data collection.
Intervention: A single intravenous administration of REGV131-LNP1265, a CRISPR/Cas9-based coagulation Factor IX gene insertion therapy, delivered across dose-escalation and dose-confirmation cohorts (Part 1) and a dose-expansion cohort at the recommended dose (Part 2A), in this Phase 1/2 open-label study.
Comparator: None (two-part, open-label, non-randomised study; no concurrent comparator arm).
Outcome: To identify a recommended dose for expansion (RDE) of REGV131-LNP1265, and to demonstrate safety and tolerability and achievement of sustained normal or near-normal plasma FIX functional activity.

0 recruited (UK total, all sites to date) 11 open sites

View open sites (11)

Barts Health NHS Trust – The Royal London Hospital
Cambridge University Hospitals NHS Foundation Trust – Addenbrooke's Hospital
Greater Glasgow and Clyde – Gartnavel Royal Hospital
Greater Glasgow and Clyde – Glasgow Royal Infirmary
Guy's and St Thomas' NHS Foundation Trust – St Thomas' Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
Lewisham and Greenwich NHS Trust – Queen Elizabeth Hospital
Oxford University Hospitals NHS Foundation Trust – Oxford University Hospitals NHS Foundation Trust
Royal Free London NHS Foundation Trust – Royal Free Hospital
University Hospital Southampton NHS Foundation Trust – Southampton General Hospital
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital

ClinicalTrials.gov →

Safety and Efficacy of GL-2045 in Patients with Immune Thrombocytopenic PurpuraInitial Safety and Efficacy, Sequential Group, Adaptive Dose Study of GL-2045 Subcutaneous Injection Repeat Doses in Patients with Immune Thrombocytopenic Purpura

Interventional Commercial Bleeding Disorders Phase I

Population: Adults (male or female) aged 18-80 with immune thrombocytopenic purpura (ITP) and a platelet count of 10-49 x10^9/L at baseline, who have previously responded to corticosteroids or IVIg as documented by a consultant haematologist.
Intervention: Repeat subcutaneous injections of GL-2045 given in a sequential-group, adaptive dose-escalation design (Phase I safety and efficacy study).
Comparator: None (single-arm, non-randomised, sequential-group dose-escalation study; no comparator arm described in the title or eligibility criteria).
Outcome: Not specified in the NIHR record.

3 recruited (UK total, all sites to date) 3 open sites

View open sites (3)

Leeds Teaching Hospitals NHS Trust – St James's University Hospital
Lothian – Royal Infirmary of Edinburgh at Little France
Oxford University Hospitals NHS Foundation Trust – Churchill Hospital

Study evaluating joint health in people with haemophilia A being treated with efanesoctocog alfaA low-interventional study to evaluate long-term effectiveness of real-world prophylactic treatment with efanesoctocog alfa on joint health in people with haemophilia A (ALTITUDE)

Interventional Commercial Bleeding Disorders Phase IV

Population: Patients of any age and either sex with a diagnosis of haemophilia A of any severity, prescribed efanesoctocog alfa prophylactic treatment within 6 months prior to enrolment (or at the enrolment visit), who received prophylactic treatment with any haemophilia product for at least 12 months beforehand, with documented pre-study bleeding and treatment data.
Intervention: Real-world, low-interventional prophylactic treatment with efanesoctocog alfa, with joint health monitored via the Haemophilia Joint Health Score (HJHS), patient-reported outcomes, and, in a HEAD-US subgroup, annual ultrasound of index joints (ankles, elbows, knees).
Comparator: None (non-randomised, low-interventional real-world study) — documented pre-study treatment and bleeding data from the 12 months prior to efanesoctocog alfa prescription are collected as historical context rather than as a formal concurrent comparator arm.
Outcome: Not specified in the NIHR record.

48 recruited (UK total, all sites to date) 6 open sites

View open sites (6)

Barts Health NHS Trust – The Royal London Hospital
Cardiff & Vale University LHB – University Hospital of Wales
Great Ormond Street Hospital for Children NHS Foundation Trust – Great Ormond Street Hospital Central London Site
Manchester University NHS Foundation Trust – Manchester Royal Infirmary
Royal Free London NHS Foundation Trust – Royal Free Hospital
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital

NOVEL-2Collection of Venous and Capillary Blood Samples For The Research, Optimisation and Calibration of New Diagnostic Devices 2.

Interventional Commercial Bleeding Disorders

Population: Adults ≥18 years attending a definitive care team with research capabilities, presenting with symptoms indicative of a thromboembolic event (Group A), infection or inflammation (Group B), cardiovascular disease/heart failure/acute coronary syndrome (Group C), renal disorders (Group D), or other presentations not fitting these cohorts (Group E).
Intervention: Collection of venous and capillary blood samples to research, develop, optimise, and calibrate new diagnostic biomarker tests on the LumiraDx point-of-care platform across embolism, infection/inflammation, cardiovascular, and renal disease biomarker categories.
Comparator: None (single-arm, open-label) — the record's randomisation field is shown as "-"; new LumiraDx assay results are benchmarked against approved gold-standard reference methods rather than a separate patient comparator arm.
Outcome: The stated aim is to research and develop new diagnostic biomarkers on the LumiraDx platform that are comparable to approved gold-standard reference methods, intended to enhance clinicians' and patients' ability to monitor health conditions and improve outcomes via near-patient, point-of-care testing.

17,579 recruited (UK total, all sites to date) 13 open sites

View open sites (13)

Greater Glasgow and Clyde – Glasgow Royal Infirmary
Greater Glasgow and Clyde – Greater Glasgow and Clyde
Greater Glasgow and Clyde – Queen Elizabeth University Hospital
Greater Glasgow and Clyde – Royal Alexandra Hospital
Lanarkshire – Lanarkshire
Lanarkshire – University Hospital Monklands
Lothian – Lothian
Lothian – Royal Infirmary of Edinburgh at Little France
Lothian – St John's Hospital
Manchester University NHS Foundation Trust – Manchester University NHS Foundation Trust
NHS National Waiting Times Centre Board – Golden Jubilee National Hospital
NHS National Waiting Times Centre Board – NHS National Waiting Times Centre Board
Northern Care Alliance NHS Foundation Trust – Northern Care Alliance NHS Foundation Trust

Genotyping and platelet phenotyping (GAPP)Mild bleeding disorders caused by platelet defects

Observational Academic / non-commercial Bleeding Disorders

This observational study (Genotyping and Platelet Phenotyping, GAPP) recruits patients aged 0 to 85 years with a platelet disorder of unknown cause, reflecting recognition that platelet function disorders are under-diagnosed, often asymptomatic outside of a bleeding challenge such as surgery or injury, and difficult to characterise with standard molecular biology approaches. The study combines clinical/laboratory platelet phenotyping with targeted gene sequencing to test the hypothesis that some patients with excessive bleeding have a previously unrecognised platelet function impairment, and investigates the functional effect of selected missense mutations through expression studies in immortalised cell lines. The purpose is to identify novel gene defects underlying platelet dysfunction, informing understanding of normal platelet physiology and diagnosis of mild bleeding disorders.

1,160 recruited (UK total, all sites to date) 33 open sites

View open sites (33)

Birmingham Women's NHS Foundation Trust – Birmingham Women's Hospital
Birmingham Women's and Children's NHS Foundation Trust – Birmingham Children's Hospital
Birmingham Women's and Children's NHS Foundation Trust – Birmingham Women's Hospital
Bristol NHS Foundation Trust – Bristol Royal Infirmary
Cardiff & Vale University LHB – Cardiff & Vale University LHB
Cardiff & Vale University LHB – University Hospital of Wales
Central Manchester University Hospitals NHS Foundation Trust – Manchester Royal Infirmary
East Kent Hospitals University NHS Foundation Trust – Kent & Canterbury Hospital
Great Ormond Street Hospital for Children NHS Foundation Trust – Great Ormond Street Hospital Central London Site
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Heart of England NHS Foundation Trust – Good Hope Hospital
Hull University Teaching Hospitals NHS Trust – Castle Hill Hospital
Leeds Teaching Hospitals NHS Trust – Clarendon Wing, Leeds General Infirmary
Leeds Teaching Hospitals NHS Trust – Leeds General Infirmary
Lothian – Royal Hospital for Sick Children (Edinburgh)
Lothian – Royal Infirmary of Edinburgh at Little France
Manchester University NHS Foundation Trust – Manchester Royal Infirmary
Non-NHS Activity In West Midlands LCRN – University of Birmingham
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - City Campus
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - Queen's Medical Centre Campus
Oxford University Hospitals NHS Foundation Trust – Churchill Hospital
Royal Devon University Healthcare NHS Foundation Trust – Royal Devon & Exeter Hospital (Wonford)
Royal Liverpool and Broadgreen University Hospitals NHS Trust – The Royal Liverpool University Hospital
Sheffield Children's NHS Foundation Trust – Sheffield Children's Hospital
Sheffield Teaching Hospitals NHS Foundation Trust – Royal Hallamshire Hospital
The Dudley Group NHS Foundation Trust – Russells Hall Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – Freeman Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary
United Lincolnshire Teaching Hospitals NHS Trust – Lincoln County Hospital
University College London Hospitals NHS Foundation Trust – National Hospital for Neurology & Neurosurgery - Queen Square
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital
University Hospitals Coventry and Warwickshire NHS Trust – University Hospital (Coventry)
University Hospitals of Leicester NHS Trust – Leicester Royal Infirmary

NIHR public summary →

Inherited fibrinogen disordersEvaluating the effects of concentrated fibrinogen replacement therapies in the management of inherited fibrinogen disorders.

Observational Academic / non-commercial Bleeding Disorders

This is an observational study of adult patients (aged 18 years or older), registered at the Oxford Haemophilia & Thrombosis Centre, with a diagnosed inherited fibrinogen disorder (afibrinogenaemia, hypofibrinogenaemia, or dysfibrinogenaemia). It evaluates, in laboratory testing of patients' plasma, the effects of two concentrated fibrinogen replacement products (Riastap and Fibryga) and cryoprecipitate on clot formation and clot stability, building on prior work suggesting that different fibrinogen products can affect clot quality differently. The aim is to give clinicians better evidence to guide treatment choice according to an individual patient's bleeding and thrombosis risk, which is particularly complex in dysfibrinogenaemia and in the peri-operative setting, where the balance of bleeding versus thrombosis risk is often unclear. There are no specific exclusion criteria.

15 recruited (UK total, all sites to date) 1 open site

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Oxford University Hospitals NHS Foundation Trust – Nuffield Orthopaedic Centre

NIHR public summary →

Protect-NOW – Octapharma’s FVIII in PUPs and MTPsPRactical utilization of Octapharma FVIII Concentrates in Previously Untreated and Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment (with Nuwiq, Octanate or Wilate). A Real-World Efficacy and Safety Observational Study

Observational Commercial Bleeding Disorders

Protect-NOW is a real-world, observational efficacy and safety study of Octapharma's factor VIII (FVIII) concentrates (Nuwiq, Octanate, or Wilate) in male and female patients of any age and ethnicity with severe haemophilia A (FVIII:C <1%) entering routine clinical treatment. Eligible patients are either previously untreated (PUPs) or minimally treated (<5 exposure days, first exposure day after 1 January 2015, with no prior inhibitor development, or continuing on the same Octapharma product after inhibitor development), for whom a decision to prescribe an Octapharma FVIII concentrate has already been made before enrolment. Patients with other coagulation disorders, on systemic immunosuppressants, or participating in interventional or other non-interventional Octapharma studies are excluded.

18 recruited (UK total, all sites to date) 4 open sites

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Birmingham Women's and Children's NHS Foundation Trust – Birmingham Children's Hospital
Great Ormond Street Hospital for Children NHS Foundation Trust – Great Ormond Street Hospital Central London Site
Oxford University Hospitals NHS Foundation Trust – John Radcliffe Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary

The CACHE Study v1.0The CACHE Study: Coronary Artery Care in HaEmophilia

Observational Academic / non-commercial Bleeding Disorders

This observational study uses a novel photon-counting detector cardiac CT scanner, combined with artificial intelligence analysis, to characterise coronary artery plaque type, burden, and rupture risk in men with haemophilia A or B (factor VIII/IX <40%) aged 45 years or older, comparing findings with people without haemophilia. It is motivated by the observation that people with haemophilia have a 40% lower risk of dying from heart disease, and aims to understand whether reduced blood clotting protects against plaque-related coronary events, with a view to informing future cardiovascular prevention and treatment strategies. Participants must be able to undergo CT imaging and give informed consent; those with contraindications to CT contrast or significant renal impairment (eGFR <30 ml/min) are excluded.

84 recruited (UK total, all sites to date) 1 open site

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Oxford University Hospitals NHS Foundation Trust – Nuffield Orthopaedic Centre

NIHR public summary →

Treatment of Hemophilia A Patients With FVIII InhibitorsMOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A – An International Low-Interventional Pragmatic Investigator Initiated Trial

Observational Academic / non-commercial Bleeding Disorders

MOTIVATE is a low-interventional, non-randomised, investigator-initiated study evaluating current approaches to managing inhibitors in patients with haemophilia A of any age and severity who have developed FVIII inhibitors (historical titre >=0.6 BU/mL), including those who have failed prior immune tolerance induction (ITI) attempts. All treatment is at the investigator's discretion, spanning three approaches: ITI with plasma-derived or recombinant FVIII products (Nuwiq, octanate, or wilate) alone; the same ITI combined with emicizumab prophylaxis; or routine emicizumab prophylaxis without ITI, with aPCC/rFVIIa available as needed to treat bleeds or cover surgery. Participants are male, not randomised to a group, and may switch groups during follow-up; the study plans to enrol 120 patients followed for up to 5 years, including sub-studies on long-term joint/bone health and thrombosis risk, to document ITI approaches and evaluate their efficacy and safety.

5 recruited (UK total, all sites to date) 5 open sites

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Great Ormond Street Hospital for Children NHS Foundation Trust – Great Ormond Street Hospital Central London Site
Manchester University NHS Foundation Trust – Royal Manchester Children's Hospital
Oxford University Hospitals NHS Foundation Trust – John Radcliffe Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – Freeman Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary

ClinicalTrials.gov →NIHR public summary →

Blood and Bone Marrow Function

3 studies

Defining the mechanisms underlying impaired fibrinolysis in antiphospholipid syndromeDefining the mechanisms underlying impaired fibrinolysis in antiphospholipid syndrome

Observational Academic / non-commercial Open to new sites Blood and Bone Marrow Function

This prospective observational study examines endothelial function in patients with haematological or immunological diseases such as antiphospholipid syndrome, systemic lupus erythematosus and rheumatoid arthritis — conditions associated with endothelial dysfunction and increased risk of thrombotic and vascular events — in comparison with healthy controls. Eligible participants are adults aged 18 or over with a haematology or immunological disease (plus healthy controls) who are able to provide informed written consent. The study combines blood measurements with non-invasive assessment of endothelial function (using EndoPAT) at presentation and after therapy or observation, with follow-up allowing correlation of findings with clinical events such as new or recurrent thrombosis or bleeding. The purpose is to determine whether existing therapies have undocumented beneficial effects on endothelial function, and whether treatment could be more effective and safer if directed at modifying endothelial function.

155 recruited (UK total, all sites to date) 1 open site

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Imperial College Healthcare NHS Trust – Hammersmith Hospital

NIHR public summary →

Evaluation of platelet function in MPNs (MPN-PLATE)Platelet function testing using multiple electrode aggregometry in patients with myeloproliferative neoplasms on antiplatelet medications.

Observational Academic / non-commercial Blood and Bone Marrow Function

This observational study investigates platelet function, using multiple electrode aggregometry on whole blood samples, in patients with myeloproliferative neoplasms (essential thrombocythaemia, polycythaemia vera, or primary myelofibrosis, including pre-fibrotic/overt fibrotic phase, or MPN-unclassifiable) who are already receiving antiplatelet therapy (aspirin, clopidogrel, dipyridamole, prasugrel or ticagrelor). Eligible participants are adults over 18 with a WHO-defined MPN diagnosis already on one of these antiplatelet agents, able to give informed consent. Platelet function tests are performed at regular intervals to assess the incidence of aspirin (and other antiplatelet drug) non-response in this population — a phenomenon well described in cardiovascular disease but not widely studied in MPN — and to determine whether non-response correlates with the incidence of thrombotic events.

203 recruited (UK total, all sites to date) 1 open site

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University Hospitals of Leicester NHS Trust – Leicester Royal Infirmary

NIHR public summary →

Human Immune Cell Development in HealthHuman Immune Cell Development in Health

Observational Academic / non-commercial Blood and Bone Marrow Function

This observational study investigates how the production of immune cells in the bone marrow (haematopoiesis) is regulated in healthy people. Blood and liquid bone marrow samples are collected from healthy adult volunteers (aged over 18, free of infection, cancer, immune system disorders, haematopoietic disorders, and recent immune-modulating/anticoagulant treatment or cancer therapy) to study how immune cells develop and which genes and proteins control this process. The purpose is to use this understanding of normal immune regulation to help explain what goes wrong in disease, ultimately supporting improved treatments for patients with infections, cancer, immune and blood disorders, and those undergoing haematopoietic stem cell transplantation.

114 recruited (UK total, all sites to date) 1 open site

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The Newcastle Upon Tyne Hospitals NHS Foundation Trust – Freeman Hospital

NIHR public summary →

Haemoglobinopathies

15 studies

HIBISCUS 2A global phase 3, randomised, double-blind and placebo-controlled study evaluating the efficacy and safety of etavopivat in adolescents and adults with sickle cell disease

RCT Commercial Haemoglobinopathies Phase III

Population: Participants aged 12 years and older with a confirmed diagnosis of sickle cell disease who had 1 to 15 documented vaso-occlusive crises (VOCs) in the 12 months before screening and moderate to severe anaemia (haemoglobin >=5.0 and <=10.0 g/dL at screening).
Intervention: Etavopivat, given in this global Phase 3, randomised (2:1), double-blind study for a 52-week double-blind treatment period, with participants who complete this period eligible to enter a further 52-week etavopivat open-label extension.
Comparator: Placebo, randomised in a 2:1 ratio (etavopivat:placebo).
Outcome: Not specified in the NIHR record.

4 recruited (UK total, all sites to date) 8 open sites

View open sites (8)

Cambridge University Hospitals NHS Foundation Trust – Addenbrooke's Hospital
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Guy's and St Thomas' NHS Foundation Trust – St Thomas' Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
London North West University Healthcare NHS Trust – Central Middlesex Hospital
University College London Hospitals NHS Foundation Trust – University College Hospital
Whittington Health NHS Trust – The Whittington Hospital

ClinicalTrials.gov →

LIBRAA 52-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group, group sequential, Phase 3 study to evaluate the efficacy and safety of rilzabrutinib in patients aged 10 to 65 years with sickle-cell disease

RCT Commercial Haemoglobinopathies Phase III

Population: Patients aged 10-65 years with sickle cell disease (HbSS or HbS/β0-thalassaemia genotype) who have had 2-10 documented acute vaso-occlusive crises (VOCs) in the 12 months before screening, ECOG performance status ≤2, up-to-date vaccination status, and (if on hydroxyurea and/or L-glutamine) a stable weight-based dose for at least 3-6 months.
Intervention: Rilzabrutinib given over a 52-week treatment period in a multicentre, parallel-group, group sequential design.
Comparator: Placebo (double-blind, placebo-controlled, parallel-group design, per title).
Outcome: Not specified in the NIHR record.

1 recruited (UK total, all sites to date) 5 open sites

View open sites (5)

Cardiff & Vale University LHB – Noahs Ark Childrens Hospital for Wales
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
London North West University Healthcare NHS Trust – Central Middlesex Hospital
Manchester University NHS Foundation Trust – Manchester Royal Infirmary
Manchester University NHS Foundation Trust – Royal Manchester Children's Hospital

ClinicalTrials.gov →

GBT60121A Phase 2/3 Randomized, Multicenter Study of GBT021601 Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease

RCT Commercial Haemoglobinopathies Phase II/III

Population: Male and female participants with sickle cell disease (HbSS or HbSβ0-thalassaemia genotype) and haemoglobin 5.5-10.5 g/dL at screening. Part A enrols adults aged 18-65 years; Part B enrols adolescents and adults aged 12-65 years with 2-10 VOCs in the prior 12 months; Part C (open-label PK study) enrols paediatric participants across four cohorts spanning 6 months to <18 years. Participants on hydroxyurea and/or L-glutamine must be on a stable dose for ≥90 days.
Intervention: GBT021601 given orally: Part A assesses safety, tolerability, and efficacy in adults to determine an optimal dose; Part B assesses efficacy over 48 weeks in adults/adolescents; Part C is an open-label, single-arm pharmacokinetic and safety study of single and multiple doses in paediatric participants.
Comparator: Placebo in Part B ("efficacy of GBT021601 versus placebo"); Parts A and C are open-label without a placebo comparator (Part C explicitly "open-label single arm").
Outcome: Part A's stated aim is to evaluate the safety, tolerability, and efficacy of GBT021601 to determine an optimal dose; Part B's stated aim is to evaluate the efficacy of GBT021601 versus placebo over 48 weeks; Part C's stated aim is to evaluate the pharmacokinetics and safety of single and multiple doses of GBT021601 in paediatric participants.

2 recruited (UK total, all sites to date) 10 open sites

View open sites (10)

Barts Health NHS Trust – The Royal London Hospital
Bristol NHS Foundation Trust – Bristol Haematology & Oncology Centre
Cambridge University Hospitals NHS Foundation Trust – Addenbrooke's Hospital
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Lewisham and Greenwich NHS Trust – University Hospital Lewisham
Manchester University NHS Foundation Trust – Royal Manchester Children's Hospital
Sandwell and West Birmingham Hospitals NHS Trust – City Health Centre
University College London Hospitals NHS Foundation Trust – University College Hospital

ClinicalTrials.gov →

A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With SCDA Phase 2, Double-blind, Randomized, Placebo-Controlled, Multicenter, Dose-Finding, Efficacy, and Safety Study of Tebapivat in Participants with Sickle Cell Disease

RCT Commercial Haemoglobinopathies Phase II

Population: Individuals aged 16 years or older with a documented diagnosis of sickle cell disease (HbSS, HbSC, HbS/beta0-thalassaemia, HbS/beta+-thalassaemia, or other sickle cell syndrome variants) and haemoglobin 5.5-10.5 g/dL, on a stable hydroxyurea dose for at least 90 days if applicable.
Intervention: Tebapivat administered at multiple dose levels in this Phase 2, multicentre, dose-finding efficacy and safety study.
Comparator: Placebo (double-blind, randomised, placebo-controlled design, per the study title).
Outcome: Not specified in the NIHR record.

2 recruited (UK total, all sites to date) 4 open sites

View open sites (4)

Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Guy's and St Thomas' NHS Foundation Trust – St Thomas' Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
University College London Hospitals NHS Foundation Trust – University College Hospital

ClinicalTrials.gov →

FERVENT-1PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NONTRANSFUSION DEPENDENT B-THALASSEMIA

RCT Commercial Haemoglobinopathies Phase II

Population: Adults aged 18–60 years with a clinical diagnosis of non-transfusion-dependent β-thalassaemia (NTDT) and resultant iron overload (liver iron concentration ≥5 mg Fe/g dry weight by R2* MRI); Part A enrols participants naïve to or who have discontinued iron chelation therapy (ICT), and Part B additionally includes participants on a stable ICT dose. Participants requiring regular blood transfusions are excluded.
Intervention: Subcutaneous REGN7999 (a TMPRSS6 inhibitor) dosed Q4W in a two-part, randomised design: Part A randomises ~25 participants 2:2:1 to 600 mg, 100 mg REGN7999, or placebo; Part B randomises ~70 participants 2:2:1 to a higher dose (up to 900 mg), a lower dose, or placebo.
Comparator: Placebo, administered subcutaneously alongside active REGN7999 arms in a double-blind, placebo-controlled design.
Outcome: The study's stated purpose is to evaluate the efficacy, safety, and tolerability of subcutaneously administered REGN7999 in participants with iron overload due to NTDT.

0 recruited (UK total, all sites to date) 5 open sites

View open sites (5)

Barts Health NHS Trust – St Bartholomew's Hospital
Barts Health NHS Trust – The Royal London Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
University College London Hospitals NHS Foundation Trust – University College Hospital
University Hospital Southampton NHS Foundation Trust – Southampton General Hospital

ClinicalTrials.gov →

Safety of Anumigilimab (CSL324) in Adults with Sickle Cell DiseasePhase 2a, Multicenter, Randomized, Doubleblind, Placebo-controlled Study to Assess the Safety of Anumigilimab (CSL324) in Adults with Sickle Cell Disease

RCT Commercial Haemoglobinopathies Phase II

Population: Adults aged 18 years and older with a confirmed diagnosis of sickle cell disease (any genotype) who experienced 1 to 12 vaso-occlusive crises (VOCs) requiring a visit to a medical facility and treatment with parenteral opioids or a parenteral NSAID within the 12 months before screening, and who are either on a stable, well-tolerated hydroxyurea (HU) regimen for at least 30 days before screening or have discontinued/refused HU.
Intervention: Anumigilimab (CSL324), administered in this Phase 2a, multicentre, randomised study; specific dose and administration schedule are not given in the provided text.
Comparator: Placebo, per the randomised, double-blind, placebo-controlled design stated in the title.
Outcome: Not specified in the NIHR record.

0 recruited (UK total, all sites to date) 9 open sites

View open sites (9)

Barts Health NHS Trust – The Royal London Hospital
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
Leeds Teaching Hospitals NHS Trust – St James's University Hospital
Liverpool University Hospitals NHS Foundation Trust – Royal Liverpool University Hospital
London North West University Healthcare NHS Trust – Central Middlesex Hospital
Manchester University NHS Foundation Trust – Manchester Royal Infirmary
St George's University Hospitals NHS Foundation Trust – St George's Hospital (Tooting)
University College London Hospitals NHS Foundation Trust – University College Hospital

ClinicalTrials.gov →

CA230-1019A Phase 1/2a, First-in-human, Randomized, Double-blinded, Placebo-controlled, Dose-finding Study in Healthy Volunteers and Participants with Sickle Cell Disease to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability, pH and Food Effect, and Preliminary Efficacy of BMS-986470

RCT Commercial Haemoglobinopathies Phase I/II

Population: Healthy volunteers aged 18–55 years (Cohort A) and adults ≥18 years with sickle cell disease (genotype HbSS, HbSβ0-thalassaemia, or HbSβ+-thalassaemia) with ≥4 vaso-occlusive crises (VOCs) in the previous 12 months (or ≥2 in the previous 6 months) who have failed, cannot tolerate, or are not candidates for approved SCD therapies (Cohort B).
Intervention: BMS-986470, administered in a first-in-human, dose-finding design to evaluate pharmacokinetics, pharmacodynamics, safety, tolerability, pH and food effect, and preliminary efficacy.
Comparator: Placebo (randomised, double-blind, placebo-controlled design, as stated in the title).
Outcome: Not specified in the NIHR record

0 recruited (UK total, all sites to date) 5 open sites

View open sites (5)

Imperial College Healthcare NHS Trust – Hammersmith Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Leeds Teaching Hospitals NHS Trust – St James's University Hospital
London North West University Healthcare NHS Trust – Northwick Park Hospital
University Hospitals Sussex NHS Foundation Trust – Royal Sussex County Hospital

ClinicalTrials.gov →

REDRESS V1.0A multi-centre open randomised controlled trial to assess the effect of related haplo-donor haematopoietic stem cell transplantation versus standard of care (no transplant) on treatment failure at 24 month in adults with severe sickle cell disease.

RCT Academic / non-commercial Haemoglobinopathies

Population: Adults aged 18 years or older with severe sickle cell disease (SCD) at high risk of morbidity and mortality (e.g. prior stroke, recurrent acute chest syndrome, frequent severe pain crises, regular transfusion dependence, red cell alloantibodies limiting transfusion, intolerance of hydroxycarbamide/transfusion, or established end-organ damage), who have a confirmed haploidentical (half-matched) family donor and meet cardiac, lung, renal, and hepatic fitness criteria for transplant.
Intervention: Related haploidentical (half-matched) donor haematopoietic stem cell transplantation (haplo-SCT), using stem cells from the blood or bone marrow of a genetically half-matched family member, intended to cure SCD.
Comparator: Standard of care (no transplant) — continuation of existing SCD management such as regular blood transfusions, hydroxycarbamide, and supportive pain management — in this open-label, randomised controlled trial, with 120 participants randomised 1:1 and followed for 2 years.
Outcome: Per the title, the primary comparison is treatment failure at 24 months between haploidentical SCT and standard of care; the research summary also states the trial aims to determine whether haploidentical SCT is safe, effective, and cost-worthy (good value for money for the NHS) relative to standard care.

39 recruited (UK total, all sites to date) 11 open sites

View open sites (11)

Barts Health NHS Trust – The Royal London Hospital
Bristol NHS Foundation Trust – Bristol Haematology & Oncology Centre
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Manchester University NHS Foundation Trust – Manchester Royal Infirmary
Sandwell and West Birmingham Hospitals NHS Trust – Sandwell General Hospital
Sheffield Teaching Hospitals NHS Foundation Trust – Royal Hallamshire Hospital
St George's University Hospitals NHS Foundation Trust – St George's Hospital (Tooting)
University College London Hospitals NHS Foundation Trust – University College Hospital
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital

ClinicalTrials.gov →NIHR public summary →

Etavopivat Floral Roll-over trialAn open-label, multi-centre, rollover study to characterise long-term safety and efficacy of Etavopivat in adults and children who have sickle cell disease or thalassemia and have completed a treatment period in an Etavopivat study

Interventional Commercial Haemoglobinopathies Phase III

Population: Adults and children with sickle cell disease or thalassaemia who are ongoing participants in an etavopivat parent study, have completed at least a treatment period of that parent study, and have derived clinical benefit from etavopivat as judged by the investigator.
Intervention: Continued (rollover) open-label treatment with etavopivat following completion of a parent etavopivat study, including participants previously on stable-dose hydroxyurea, crizanlizumab or L-glutamine.
Comparator: None (open-label, non-randomised rollover study; no comparator arm).
Outcome: Not specified in the NIHR record.

7 recruited (UK total, all sites to date) 7 open sites

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Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
Imperial College Healthcare NHS Trust – St Mary's Hospital (HQ)
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Manchester University NHS Foundation Trust – Manchester Royal Infirmary
Oxford University Hospitals NHS Foundation Trust – Churchill Hospital
Sheffield Teaching Hospitals NHS Foundation Trust – Royal Hallamshire Hospital

ClinicalTrials.gov →

Haem-Match Feasibility StudyA single centred feasibility study to evaluate and compare the implementation of an antigen-prioritising algorithm with standard care to arrange units for blood transfusion prior to selection by staff at NHSBT

Interventional Academic / non-commercial Haemoglobinopathies

Population: Adults aged 16 years or older with sickle cell disorder (any genotype) on a regular exchange blood transfusion (EBT) programme, with at least three contiguous transfusion episodes and red cell genotype data submitted on the Axiom array; around 40 participants will be recruited at a single UK site. Patients who are difficult to transfuse, have poor treatment concordance, or are pregnant/planning pregnancy are excluded.
Intervention: Use of the bloodMatcher algorithm, which arranges donor blood units for selection by NHS Blood and Transplant (NHSBT) staff principally according to extended blood group compatibility rather than unit age, applied across three study exchange-transfusion episodes per participant.
Comparator: The current NHSBT algorithm, which principally arranges donor units by age (then location) prior to staff selection; each participant acts as their own control, with three standard transfusion episodes compared against three bloodMatcher-guided episodes in this non-randomised, single-arm feasibility study.
Outcome: Not specified in the NIHR record.

1 recruited (UK total, all sites to date) 1 open site

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University College London Hospitals NHS Foundation Trust – University College Hospital

NIHR public summary →

IPIG PNH RegistryInternational PNH Interest Group PNH Registry

Observational Commercial Haemoglobinopathies

The International PNH Interest Group (IPIG) PNH Registry is an observational registry for patients with paroxysmal nocturnal haemoglobinuria (PNH) confirmed by flow cytometry. Patients (or their parent/legally authorised representative) provide informed consent/assent to participate. Patients who subsequently enrol in an interventional PNH clinical trial are retained in the registry but have data collection paused during trial participation, resuming afterwards. The specific research objectives and data items collected are not detailed in the available record.

362 recruited (UK total, all sites to date) 7 open sites

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Cardiff & Vale University LHB – Cardiff & Vale University LHB
Cardiff & Vale University LHB – University Hospital of Wales
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Lanarkshire – University Hospital Monklands
Leeds Teaching Hospitals NHS Trust – St James's University Hospital
Leeds Teaching Hospitals NHS Trust – St James's University Hospital
St George's University Hospitals NHS Foundation Trust – St George's Hospital (Tooting)

LTFU for patients treated with CTX001A Long-term Follow-up Study of Subjects With β thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

Observational Commercial Haemoglobinopathies

This is a long-term follow-up study of patients with beta-thalassaemia or sickle cell disease who have previously received an infusion of CTX001, an autologous CRISPR-Cas9 gene-edited haematopoietic stem cell product. Eligibility requires signed informed consent and prior receipt of CTX001 infusion, with no exclusion criteria specified. The research summary was not provided in the record, so the specific long-term data being collected and the study's detailed purpose are not described beyond continued follow-up of these previously treated patients.

5 recruited (UK total, all sites to date) 4 open sites

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Barts Health NHS Trust – The Royal London Hospital
Great Ormond Street Hospital for Children NHS Foundation Trust – Great Ormond Street Hospital Central London Site
Imperial College Healthcare NHS Trust – Hammersmith Hospital
University College London Hospitals NHS Foundation Trust – University College Hospital

ClinicalTrials.gov →

Sickle cell lines, Version 1Development and use of cell models for Sickle Cell Disease by in vitro differentiation of primary stem cells and the generation of immortalised cell lines from sickle cell patients.

Observational Academic / non-commercial Haemoglobinopathies

This laboratory-based study collects stem cells and blood/tissue samples from patients aged 10 to 40 years with confirmed sickle cell disease (HbSS, HbSC, HbS/beta thalassaemia, HbSOman, or HbSDPunjab genotypes) and from healthy donors without a haemoglobinopathy, to generate in vitro-differentiated red blood cells and immortalised cell lines. The aim is to study the altered properties of sickle red blood cells compared with healthy cells, use these cell models to investigate the mechanisms underlying red cell defects in SCD, test new treatments, and explore the genetic basis of disease severity. Patients who have had a recent blood transfusion, alpha thalassaemia, G6PD deficiency, or a previous bone marrow transplant are excluded from blood sampling.

23 recruited (UK total, all sites to date) 1 open site

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King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)

NIHR public summary →

The Improving Black Health Outcomes BioResource (IBHO)Improving Black Health Outcomes

Observational Academic / non-commercial Open to new sites Haemoglobinopathies

This is an observational BioResource study recruiting people in the UK who identify as Black or of Black ancestry (including Black African, Black Caribbean, Black African-Caribbean, Black British, and related or mixed Black heritage backgrounds), as well as participants with a confirmed diagnosis of sickle cell disease or thalassaemia regardless of ethnicity. Its purpose is to research the social and biological causes and impacts of health conditions affecting Black communities, including conditions occurring at increased rates (such as sickle cell disease, diabetes, and pregnancy/childbirth complications) and conditions that are under-studied in this population (such as anxiety, depression, and dermatological problems). The BioResource aims to improve understanding of how and why these conditions occur, identify the best assessment tools for accurate diagnosis in Black people, and use findings to develop treatments meeting the specific health needs of Black communities. People unwilling to be contacted about future research or to have their data/samples shared are excluded.

6,651 recruited (UK total, all sites to date) 177 open sites

View open sites (177)

Alder Hey Children's NHS Foundation Trust – Alder Hey Children's NHS Foundation Trust
Barking, Havering and Redbridge University Hospitals NHS Trust – Queen's Hospital
Barts Health NHS Trust – Mile End Hospital
Barts Health NHS Trust – Newham General Hospital
Barts Health NHS Trust – St Bartholomew's Hospital
Barts Health NHS Trust – The Royal London Hospital
Barts Health NHS Trust – Whipps Cross University Hospital
Belfast Health and Social Care Trust – The Royal Victoria Hospital
Birmingham Women's and Children's NHS Foundation Trust – Birmingham Children's Hospital
Birmingham Women's and Children's NHS Foundation Trust – Birmingham Women's Hospital
Blackpool Teaching Hospitals NHS Foundation Trust – Blackpool Victoria Hospital
Blackpool Teaching Hospitals NHS Foundation Trust – Clifton Hospital
Blackpool Teaching Hospitals NHS Foundation Trust – Fleetwood Hospital
Bristol NHS Foundation Trust – Bristol Haematology & Oncology Centre
Bristol NHS Foundation Trust – Bristol Royal Hospital for Children
Bristol NHS Foundation Trust – Clinical Research Facility (Crf)
Bristol NHS Foundation Trust – Uhbw Clinical Research Facility - Azd2816 Covid19 Trials
Buckinghamshire Healthcare NHS Trust – Stoke Mandeville Hospital
Cambridge University Hospitals NHS Foundation Trust – Addenbrooke's Hospital
Cambridge University Hospitals NHS Foundation Trust – Leeds General Infirmary
Dartford and Gravesham NHS Trust – Medway Maritime Hospital
East Suffolk and North Essex NHS Foundation Trust – Colchester General Hospital
East and North Hertfordshire Teaching NHS Trust – Lister Hospital
Epsom and St Helier University Hospitals NHS Trust – Epsom Hospital
Epsom and St Helier University Hospitals NHS Trust – St Helier Hospital
Great Ormond Street Hospital for Children NHS Foundation Trust – Great Ormond Street Hospital Central London Site
Guy's and St Thomas' NHS Foundation Trust – Guy's Hospital
Homerton Healthcare NHS Foundation Trust – Homerton University Hospital
Hull University Teaching Hospitals NHS Trust – Castle Hill Hospital
Imperial College Healthcare NHS Trust – Charing Cross Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
Imperial College Healthcare NHS Trust – Hammersmith Hospital
Imperial College Healthcare NHS Trust – Queen Charlotte's Hospital
Imperial College Healthcare NHS Trust – St Mary's Hospital (HQ)
Imperial College Healthcare NHS Trust – Western Eye Hospital
King's College Hospital NHS Foundation Trust – King's College Hospital (Denmark Hill)
Lancashire Teaching Hospitals NHS Foundation Trust – Royal Preston Hospital
Leeds Teaching Hospitals NHS Trust – Chapel Allerton Hospital
Leeds Teaching Hospitals NHS Trust – Clarendon Wing, Leeds General Infirmary
Leeds Teaching Hospitals NHS Trust – Leeds General Infirmary
Leeds Teaching Hospitals NHS Trust – Seacroft Hospital
Leeds Teaching Hospitals NHS Trust – St James's University Hospital
Lewisham and Greenwich NHS Trust – Queen Elizabeth Hospital
Lewisham and Greenwich NHS Trust – University Hospital Lewisham
Lincolnshire Partnership NHS Foundation Trust – Gervas House
Liverpool University Hospitals NHS Foundation Trust – Royal Liverpool University Hospital
Liverpool Women's NHS Foundation Trust – Liverpool Womens Hospital
London North West University Healthcare NHS Trust – Central Middlesex Hospital
London North West University Healthcare NHS Trust – Northwick Park Hospital
Manchester University NHS Foundation Trust – Blackpool Victoria Hospital
Manchester University NHS Foundation Trust – Manchester Royal Infirmary
Manchester University NHS Foundation Trust – St Mary's Hospital
Medway NHS Foundation Trust – Medway Maritime Hospital
Mersey and West Lancashire Teaching Hospitals NHS Trust – Ormskirk and District General Hospital
Mersey and West Lancashire Teaching Hospitals NHS Trust – Southport and Formby District General Hospital
Mersey and West Lancashire Teaching Hospitals NHS Trust – Whiston Hospital
Mid and South Essex NHS Foundation Trust – Mid Essex Hospital
Mid and South Essex NHS Foundation Trust – Signpost (Basildon)
Mid and South Essex NHS Foundation Trust – The Megacentre Rayleigh
Midlands Partnership University NHS Foundation Trust – Cheadle Hospital
Midlands Partnership University NHS Foundation Trust – Haywood Hospital
Moorfields Eye Hospital NHS Foundation Trust – Moorfields Eye Hospital (City Road Campus)
NIHR RDN: East Midlands – Abington Medical Centre
NIHR RDN: East Midlands – Albany House Medical Centre
NIHR RDN: East Midlands – Billinghay Medical Practice
NIHR RDN: East Midlands – Bushloe Surgery
NIHR RDN: East Midlands – Caythorpe & Ancaster Medical Practice
NIHR RDN: East Midlands – Colsterworth Surgery
NIHR RDN: East Midlands – East Lindsey Medical Group
NIHR RDN: East Midlands – Enderby Medical Centre
NIHR RDN: East Midlands – Forest Medical Group
NIHR RDN: East Midlands – Kingsway Surgery
NIHR RDN: East Midlands – Langham Place Surgery
NIHR RDN: East Midlands – Lindum Medical Practice
NIHR RDN: East Midlands – Long Bennington Medical Centre
NIHR RDN: East Midlands – Market Cross Surgery
NIHR RDN: East Midlands – Millview Medical Centre
NIHR RDN: East Midlands – Nettleham Medical Practice
NIHR RDN: East Midlands – Ruskington Surgery
NIHR RDN: East Midlands – Sleaford Medical Group
NIHR RDN: East Midlands – South Leicestershire Medical Group
NIHR RDN: East Midlands – St. Johns Medical Centre
NIHR RDN: East Midlands – St. Peters Hill Surgery
NIHR RDN: East Midlands – The Billesdon Surgery
NIHR RDN: East Midlands – The Deepings Practice
NIHR RDN: East Midlands – The Glenside Country Practice
NIHR RDN: East Midlands – The Harrowby Lane Surgery
NIHR RDN: East Midlands – The Limes Medical Centre
NIHR RDN: East Midlands – The New Springwells Practice
NIHR RDN: East Midlands – Towcester Medical Centre
NIHR RDN: East Midlands – Vine Street Surgery
NIHR RDN: East of England – Boroughbury Medical Centre
NIHR RDN: East of England – Colchester Medical Practice
NIHR RDN: East of England – Creffield Medical Group
NIHR RDN: East of England – The Portmill Surgery
NIHR RDN: East of England – Thomas Walker Westgate Healthcare
NIHR RDN: East of England – West Street Surgery
NIHR RDN: North East and North Cumbria – Beacon View Medical Centre
NIHR RDN: North East and North Cumbria – Brunton Park
NIHR RDN: North East and North Cumbria – Denton Turret Medical Centre
NIHR RDN: North East and North Cumbria – Dilston Medical Centre
NIHR RDN: North East and North Cumbria – Elswick Family Practice
NIHR RDN: North East and North Cumbria – Holmside Medical Group
NIHR RDN: North East and North Cumbria – Newburn Surgery
NIHR RDN: North East and North Cumbria – Newcastle Medical Centre
NIHR RDN: North East and North Cumbria – Prospect Medical Centre
NIHR RDN: North East and North Cumbria – Saville Medical Group
NIHR RDN: North East and North Cumbria – Thornfield Medical Group
NIHR RDN: North East and North Cumbria – West Road Medical Centre
NIHR RDN: North West – 4 Seasons Medical Centre
NIHR RDN: North West – Abercromby Family Practice
NIHR RDN: North West – Albion Surgery
NIHR RDN: North West – Chapelford Primary Care Centre
NIHR RDN: North West – Culcheth Medical Centre
NIHR RDN: North West – Dingle Park Practice
NIHR RDN: North West – Guardian Street Med/Ctr
NIHR RDN: North West – Parkview Medical Centre
NIHR RDN: North West – Springfields Medical Centre
NIHR RDN: North West – St. James' Health Centre
NIHR RDN: North West – The Elms Medical Centre
NIHR RDN: North West – Vauxhall Health Centre
NIHR RDN: Yorkshire and Humber – Clover Group Practice
NIHR RDN: Yorkshire and Humber – Darnall Hall Surgery
NIHR RDN: Yorkshire and Humber – University Health Centre
NIHR RDN: Yorkshire and Humber – Windmill Health Centre
Norfolk and Norwich University Hospitals NHS Foundation Trust – Norfolk & Norwich University Hospital
North East London NHS Foundation Trust – Goodmayes Hospital - Sunflowers Court
North East London NHS Foundation Trust – Goodmayes Hospital - Trust Headquarters
North Middlesex University Hospital NHS Trust – North Middlesex Hospital
North Tees and Hartlepool NHS Foundation Trust – University Hospital of North Tees
Northampton General Hospital NHS Trust – Northampton General Hospital (Acute)
Northamptonshire Healthcare NHS Foundation Trust – Northampton General Hospital
Northern Care Alliance NHS Foundation Trust – Salford Royal
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - City Campus
Nottingham University Hospitals NHS Trust – Nottingham University Hospitals NHS Trust - Queen's Medical Centre Campus
Oxford Health NHS Foundation Trust – Warneford Hospital
Oxford University Hospitals NHS Foundation Trust – Churchill Hospital
Oxford University Hospitals NHS Foundation Trust – John Radcliffe Hospital
Oxford University Hospitals NHS Foundation Trust – Nuffield Orthopaedic Centre
Oxleas NHS Foundation Trust – Pinewood House
Rotherham Doncaster and South Humber NHS Foundation Trust – Doncaster - Tickhill Road Site
Royal Devon University Healthcare NHS Foundation Trust – Royal Devon & Exeter Hospital (Wonford)
Royal Free London NHS Foundation Trust – North Middlesex Hospital
Royal Free London NHS Foundation Trust – Royal Free Hospital
Royal Papworth Hospital NHS Foundation Trust – Royal Papworth Hospital (Papworth Everard)
Royal Surrey County Hospital NHS Foundation Trust – Royal Surrey County Hospital
Sandwell and West Birmingham Hospitals NHS Trust – City Hospital
Sheffield Children's NHS Foundation Trust – Sheffield Children's Hospital
Sheffield Teaching Hospitals NHS Foundation Trust – Darnall Primary Care Centre
Sheffield Teaching Hospitals NHS Foundation Trust – Northern General Hospital
Sheffield Teaching Hospitals NHS Foundation Trust – Royal Hallamshire Hospital
Shropshire Community Health NHS Trust – Occupational Health Gains Park
Shropshire Community Health NHS Trust – Shropshire Community Health - Coral House
Somerset NHS Foundation Trust – Musgrove Park Hospital
South Tees Hospitals NHS Foundation Trust – The James Cook University Hospital
South West London and St George's Mental Health NHS Trust – Springfield University Hospital
St George's University Hospitals NHS Foundation Trust – St George's Hospital (Tooting)
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – Freeman Hospital
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary
The Royal Marsden NHS Foundation Trust – The Royal Marsden Hospital (London)
The Royal Marsden NHS Foundation Trust – The Royal Marsden Hospital (Surrey)
The Walton Centre NHS Foundation Trust – The Walton Centre NHS Foundation Trust
United Lincolnshire Teaching Hospitals NHS Trust – Lincoln County Hospital
United Lincolnshire Teaching Hospitals NHS Trust – Pilgrim Hospital
University College London Hospitals NHS Foundation Trust – University College Hospital
University College London Hospitals NHS Foundation Trust – University College London
University Hospital Southampton NHS Foundation Trust – Southampton General Hospital
University Hospitals Birmingham NHS Foundation Trust – Queen Elizabeth Hospital
University Hospitals of Derby and Burton NHS Foundation Trust – Royal Derby Hospital
University Hospitals of Leicester NHS Trust – Glenfield Hospital
University Hospitals of Leicester NHS Trust – Leicester General Hospital
University Hospitals of Leicester NHS Trust – Leicester Royal Infirmary
University Hospitals of North Midlands NHS Trust – Cheadle Hospital
West Hertfordshire Teaching Hospitals NHS Trust – Hemel Hempstead Hospital
West Hertfordshire Teaching Hospitals NHS Trust – St Albans City Hospital
West Hertfordshire Teaching Hospitals NHS Trust – Watford General Hospital
Whittington Health NHS Trust – The Whittington Hospital

NIHR public summary →

Vasculopathy Induced Injury in SCD - 7 Tesla MRI Assessment (VISTA)Developing Novel Biomarkers of Vasculopathy-Induced Brain Injury in Childhood Sickle Cell Disease Using Ultra High Field MRI - VISTA Study

Observational Academic / non-commercial Haemoglobinopathies

This prospective, non-randomised observational study (VISTA) uses ultra-high field 7 Tesla MRI to develop novel biomarkers of vasculopathy-induced brain injury in childhood sickle cell disease (SCD), motivated by the high rate of silent cerebral infarcts (~40% by age 18) and the limited sensitivity of conventional imaging to small-vessel disease. It will recruit 33 children aged 7-16 years: 22 with confirmed SCD (Group A with imaging manifestations of SCD or abnormal TCD velocities, and Group B on close monitoring for secondary stroke prevention due to prior stroke or severe vasculopathy) and 11 age-matched healthy controls (Group C). 7T MRI data will be combined with 3T MRI and routine clinical data, with the aim of generating preliminary data to support larger biomarker-development studies and eventually identify early disease markers for future therapeutic trials.

10 recruited (UK total, all sites to date) 1 open site

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Guy's and St Thomas' NHS Foundation Trust – St Thomas' Hospital

NIHR public summary →

Thrombosis

2 studies

START UK Pilot TrialA feasibility randomised controlled trial assessing the use of platelet transfusions versus modified dose anticoagulation in patients with thrombocytopaenia and cancer-associated Thrombosis receiving anticoagulation (START UK)

RCT Academic / non-commercial Thrombosis

Population: Adults aged 18 years or older with active malignancy and imaging-confirmed pulmonary embolism, deep vein thrombosis, or unusual-site venous thromboembolism within the preceding 14 days requiring anticoagulation, with a platelet count below 50x10^9/L attributable to cancer therapy or the malignancy itself.
Intervention: Modified-dose low molecular weight heparin (LMWH) without platelet transfusion support, with dosing adjusted by platelet count (50% dose if 25-50x10^9/L; anticoagulation held if <25x10^9/L).
Comparator: Platelet transfusion support (given for 14 days when platelet count is <50x10^9/L) combined with a higher LMWH dose after transfusion, compared against the no-transfusion modified-dose LMWH arm in this multicentre, non-blinded, randomised feasibility trial.
Outcome: Primary objective: to determine the feasibility of designing and recruiting to a full-scale randomised controlled trial of management in patients with acute cancer-associated thrombosis and thrombocytopenia. Secondary objectives include additional feasibility measures (recruitment rate, reasons for non-participation, withdrawal rates), duration of thrombocytopenia, number of platelet/red cell units transfused, and feasibility of collecting EQ-5D-5L quality-of-life data.

12 recruited (UK total, all sites to date) 5 open sites

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Oxford University Hospitals NHS Foundation Trust – Churchill Hospital
Oxford University Hospitals NHS Foundation Trust – John Radcliffe Hospital
Oxford University Hospitals NHS Foundation Trust – Nuffield Orthopaedic Centre
The Newcastle Upon Tyne Hospitals NHS Foundation Trust – The Royal Victoria Infirmary
University College London Hospitals NHS Foundation Trust – University College Hospital

ISRCTN Registry →NIHR public summary →

AtenativA multicentre, prospective, open-label, uncontrolled Phase 3 study to assess the efficacy, safety and pharmacokinetics of Atenativ in patients with congenital antithrombin deficiency undergoing surgery or delivery

Interventional Commercial Thrombosis Phase III

Population: Adults aged 18-80 with documented congenital antithrombin deficiency (plasma antithrombin <=60%) and a personal or family history of thromboembolic events, who are either non-pregnant surgical patients scheduled for an elective procedure with high thromboembolic risk, or pregnant patients at >=27 weeks gestation scheduled for caesarean section or delivery.
Intervention: Administration of Atenativ (antithrombin concentrate) during the surgical or peripartum period, in this multicentre, open-label Phase 3 study assessing efficacy, safety and pharmacokinetics.
Comparator: None (open-label, uncontrolled, non-randomised study, per the title).
Outcome: Not specified in the NIHR record.

2 recruited (UK total, all sites to date) 3 open sites

View open sites (3)

Guy's and St Thomas' NHS Foundation Trust – St Thomas' Hospital
Royal Free London NHS Foundation Trust – Royal Free Hospital
University College London Hospitals NHS Foundation Trust – University College Hospital

ClinicalTrials.gov →

Anaemias

TIARAProduction of expanded autologous regulatory T cells to treat patients with aplastic anaemia: A phase I study

Alexion PNH Registry M07-001Paroxysmal Noctural Hemoglobinuria (PNH) Registry

Ascelus-HA multi-centre investigation of use of a digital follow-up pathway utilising the Ascelus app for monitoring long-term blood conditions, incorporating patient, carer, and clinician co-design and evaluation, clinical validation, and safety investigations

COMPLETEA single arm, multicentre observational study to evaluate effectiveness of pegcetacoplan under real world conditions in patients with paroxysmal nocturnal hemoglobinuria (PNH)

INR-CALCollection Of Venous And Capillary Blood For Development And Support Of The LumiraDx INR Assay

The Molecular Investigation of Unexplained AnaemiasThe Molecular Investigation of Unexplained Anaemias and Related Congenital Anaemias

Autoimmune Disorders

ARGX-113-2402A Phase 3, Multicenter, Randomized, Double-Blinded, PlaceboControlled, Parallel-Arm Study Followed by an Open-Label Arm to Evaluate the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia

Meza ITP StudyA Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of Mezagitamab Subcutaneous Injection in Participants with Persistent or Chronic Primary Immune Thrombocytopenia

Ph 3, Mavorixafor in Participants w/ Congenital or Acquired Primary Autoimmune or Idiopathic CNDA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of Mavorixafor in Participants with Congenital or Acquired Primary Autoimmune or Idiopathic Chronic Neutropenic Disorders

Study of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell DisordersA Phase 2a, Open-Label, Multicenter Study of Tafasitamab in Adult Participants With Primary Autoimmune Blood Cell Disorders

Study to Investigate the Safety and Efficacy of Pirtobrutinib in Adults with Immune ThrombocytopeniaA Phase 1/2, Dose-finding Study Investigating the Safety and Efficacy of Pirtobrutinib in Adults with Immune Thrombocytopenia

APL-3007Safety and Tolerability of APL-3007 Administered as a Single Dose in Addition to Background Therapy with a C5 Inhibitor in Adults with Paroxysmal Nocturnal Hemoglobinuria

GUMBITA pilot study to investigate the gut microbiome in patients with immune thrombocytopenia (ITP)

UK TTP RegistryUnited Kingdom Thrombotic Thrombocytopenic Purpura Registry: A prospective study of Thrombotic Thrombocytopenic Purpura in the UK

UKAITPRUnited Kingdom Adult Idiopathic Thrombocytopenic Purpura (ITP) Registry: An Investigation of Disease Progression, Treatment Effectiveness, and Co-morbid Conditions

Bleeding Disorders

Type III VWD (IS)A PHASE III, MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF EMICIZUMAB PROPHYLAXIS IN PATIENTS WITH TYPE 3 VON WILLEBRAND DISEASE

HMB-001-201A Clinical Study to Assess the Safety and Efficacy of Sutacimig in Participants with Congenital Factor VII Deficiency

Safety, tolerability, PK, PD, and efficacy of HMB-002 in participants with Von Willebrand DiseaseA Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-002 in Participants with Von Willebrand Disease

VOYAGER 2Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Peroral Doses of Inno8 in People with Haemophilia A

VIVID-6A Prospective, Multicenter, Open-label, Phase 3 Clinical Study to Evaluate the Efficacy and Safety of Prophylactic VGA039 in Adolescent and Adult Patients with von Willebrand Disease

BEYOND-9A Two-Part Open-Label Study of REGV131-LNP1265, a CRISPR/Cas9-Based Coagulation Factor IX Gene Insertion Therapy in Participants with Hemophilia B

Safety and Efficacy of GL-2045 in Patients with Immune Thrombocytopenic PurpuraInitial Safety and Efficacy, Sequential Group, Adaptive Dose Study of GL-2045 Subcutaneous Injection Repeat Doses in Patients with Immune Thrombocytopenic Purpura

Study evaluating joint health in people with haemophilia A being treated with efanesoctocog alfaA low-interventional study to evaluate long-term effectiveness of real-world prophylactic treatment with efanesoctocog alfa on joint health in people with haemophilia A (ALTITUDE)

NOVEL-2Collection of Venous and Capillary Blood Samples For The Research, Optimisation and Calibration of New Diagnostic Devices 2.

Genotyping and platelet phenotyping (GAPP)Mild bleeding disorders caused by platelet defects

Inherited fibrinogen disordersEvaluating the effects of concentrated fibrinogen replacement therapies in the management of inherited fibrinogen disorders.

Protect-NOW – Octapharma’s FVIII in PUPs and MTPsPRactical utilization of Octapharma FVIII Concentrates in Previously Untreated and Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment (with Nuwiq, Octanate or Wilate). A Real-World Efficacy and Safety Observational Study

The CACHE Study v1.0The CACHE Study: Coronary Artery Care in HaEmophilia

Treatment of Hemophilia A Patients With FVIII InhibitorsMOdern Treatment of Inhibitor-PositiVe PATiEnts with Haemophilia A – An International Low-Interventional Pragmatic Investigator Initiated Trial

Blood and Bone Marrow Function

Defining the mechanisms underlying impaired fibrinolysis in antiphospholipid syndromeDefining the mechanisms underlying impaired fibrinolysis in antiphospholipid syndrome

Evaluation of platelet function in MPNs (MPN-PLATE)Platelet function testing using multiple electrode aggregometry in patients with myeloproliferative neoplasms on antiplatelet medications.

Human Immune Cell Development in HealthHuman Immune Cell Development in Health

Haemoglobinopathies

HIBISCUS 2A global phase 3, randomised, double-blind and placebo-controlled study evaluating the efficacy and safety of etavopivat in adolescents and adults with sickle cell disease

LIBRAA 52-week, multicenter, randomized, double-blind, placebo-controlled, parallel-group, group sequential, Phase 3 study to evaluate the efficacy and safety of rilzabrutinib in patients aged 10 to 65 years with sickle-cell disease

GBT60121A Phase 2/3 Randomized, Multicenter Study of GBT021601 Administered Orally to Participants with Sickle Cell Disease and an Open-Label Pharmacokinetics Study in Pediatric Participants with Sickle Cell Disease

A Dose-Finding Study of Tebapivat to Assess Efficacy, and Safety in Participants With SCDA Phase 2, Double-blind, Randomized, Placebo-Controlled, Multicenter, Dose-Finding, Efficacy, and Safety Study of Tebapivat in Participants with Sickle Cell Disease

FERVENT-1PHASE 2, TWO-PART, RANDOMIZED, DOUBLE-BLIND, PLACEBOCONTROLLED, MULTICENTER STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF SUBCUTANEOUSLY ADMINISTERED REGN7999 (AN INHIBITOR OF TMPRSS6) IN PARTICIPANTS WITH IRON OVERLOAD DUE TO NONTRANSFUSION DEPENDENT B-THALASSEMIA

Safety of Anumigilimab (CSL324) in Adults with Sickle Cell DiseasePhase 2a, Multicenter, Randomized, Doubleblind, Placebo-controlled Study to Assess the Safety of Anumigilimab (CSL324) in Adults with Sickle Cell Disease

REDRESS V1.0A multi-centre open randomised controlled trial to assess the effect of related haplo-donor haematopoietic stem cell transplantation versus standard of care (no transplant) on treatment failure at 24 month in adults with severe sickle cell disease.

Etavopivat Floral Roll-over trialAn open-label, multi-centre, rollover study to characterise long-term safety and efficacy of Etavopivat in adults and children who have sickle cell disease or thalassemia and have completed a treatment period in an Etavopivat study

Haem-Match Feasibility Study​​​​​​​​A single centred feasibility study to evaluate and compare the implementation of an antigen-prioritising algorithm with standard care to arrange units for blood transfusion prior to selection by staff at NHSBT

IPIG PNH RegistryInternational PNH Interest Group PNH Registry

LTFU for patients treated with CTX001A Long-term Follow-up Study of Subjects With β thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)

Sickle cell lines, Version 1Development and use of cell models for Sickle Cell Disease by in vitro differentiation of primary stem cells and the generation of immortalised cell lines from sickle cell patients.

The Improving Black Health Outcomes BioResource (IBHO)Improving Black Health Outcomes

Vasculopathy Induced Injury in SCD - 7 Tesla MRI Assessment (VISTA)Developing Novel Biomarkers of Vasculopathy-Induced Brain Injury in Childhood Sickle Cell Disease Using Ultra High Field MRI - VISTA Study

Thrombosis

START UK Pilot TrialA feasibility randomised controlled trial assessing the use of platelet transfusions versus modified dose anticoagulation in patients with thrombocytopaenia and cancer-associated Thrombosis receiving anticoagulation (START UK)

AtenativA multicentre, prospective, open-label, uncontrolled Phase 3 study to assess the efficacy, safety and pharmacokinetics of Atenativ in patients with congenital antithrombin deficiency undergoing surgery or delivery

HaemTRIAL

HaemTRIAL@gmail.com

Haem-Match Feasibility StudyA single centred feasibility study to evaluate and compare the implementation of an antigen-prioritising algorithm with standard care to arrange units for blood transfusion prior to selection by staff at NHSBT