UK Haematology Trials: Coming Soon (In Setup)
Studies on the NIHR CRN Haematology portfolio that are in setup and not yet open to recruitment. Listed so the community can see what's coming.
Autoimmune Disorders
3 studiesROMISTER: Phase 3 Study of Romiplostim in Adults With Previously Untreated ITPA Phase 3, Randomized, Multicenter, Open-label Study to Evaluate the Efficacy and Safety of Romiplostim Plus Predniso(lo)ne vs. Predniso(lo)ne Alone for the Treatment of Adults With Previously Untreated Primary Immune Thrombocytopenia (ITP)
No summary was provided in the NIHR record for this study.
Study of Zola-cel (BMS-986353), CD19 NEX-T CAR T Cells in Autoimmune Cytopenia (Breakfree-AiCE)A Phase 2, Multicenter, Open-Label Study of Zolacabtagene Autoleucel (BMS-986353), CD19- Targeted NEX-T CAR T Cells, in Participants with Chronic Immune Thrombocytopenia (cITP) and Autoimmune Hemolytic Anemia (AIHA)
No summary was provided in the NIHR record for this study.
CRSP-AID-501A Phase 1/2 Dose Evaluation Trial of the Safety and Preliminary Efficacy of Anti-CD19 Allogeneic CRISPR-Cas9–Engineered T Cells (CTX112) in Adult Participants With Relapsed/Refractory Hematologic Autoimmune Disease
No summary was provided in the NIHR record for this study.
Bleeding Disorders
5 studiesHMBeacon: Study to Evaluate ALN-6400 in Adult & Adolescent Female Patients with VWD and HMBHMBeacon: A Phase 2, Randomized, Double-blind Study of the Safety, Tolerability, Efficacy, and Pharmacodynamics of Multiple Dose ALN-6400 in Adult and Adolescent Female Patients with Von Willebrand Disease (VWD) and Heavy Menstrual Bleeding (HMB)
No summary was provided in the NIHR record for this study.
A Phase 3 Study of Safety and Effectiveness of GF and FE in Haemophilia A and BA PHASE 3, NON-INVESTIGATIONAL PRODUCT, MULTI COUNTRY COHORT STUDY TO DESCRIBE THE LONG-TERM SAFETY AND EFFECTIVENESS OF A PRIOR SINGLE DOSE TREATMENT WITH INVESTIGATIVE GIROCTOCOGENE FITELPARVOVEC OR FIDANACOGENE ELAPARVOVEC IN PARTICIPANTS WITH HEMOPHILIA A OR HEMOPHILIA B, RESPECTIVELY
No summary was provided in the NIHR record for this study.
FRONTIER AHAA multinational, open-label study to investigate efficacy and safety of denecimig (Mim8) in adults with acquired haemophilia A
No summary was provided in the NIHR record for this study.
Identifying and treating bleeds while using novel haemophilia TxHow does the use of novel therapeutics with enhanced haemostatic protection influence symptoms, decision-making processes, and self-management when treating a bleed in people with haemophilia A? A qualitative study
- Qualitative study (focus groups, analysed by the Framework Analysis approach) exploring how people with haemophilia A and their treating clinicians make bleed-treatment decisions in the era of extended half-life factor, non-factor and gene therapies.
- Patient group: adults treated for a bleed in the past 12 months across a range of regimens -- severe/moderate HA on extended half-life rFVIII or emicizumab prophylaxis, severe HA post-gene therapy, or mild HA (FVIII 6-20 IU/dL) on on-demand treatment for a joint bleed.
- Clinician group: haemophilia HCPs (haematologists, nurses, physiotherapists) with 3+ years' experience routinely assessing and treating musculoskeletal bleeding.
- UK haemophilia treatment centre registration required; excludes haemophilia B and those unable to speak/understand English.
Qualitative interviews to understand patient experiences with hemophilia gene therapyQualitative interviews to understand patient experiences with hemophilia gene therapy
No summary was provided in the NIHR record for this study.
Haemoglobinopathies
1 studyVX21-CTX001-171A Phase 3 Study to Evaluate Efficacy and Safety of a Single Dose of Exa-cel in Subjects with Severe Sickle Cell Disease, βS/βC Genotype
No summary was provided in the NIHR record for this study.
Thrombosis
2 studiesCrovalimab in APSA PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER STUDY EVALUATING EFFICACY, SAFETY, AND PHARMACOKINETICS OF CROVALIMAB IN PATIENTS WITH ANTIPHOSPHOLIPID SYNDROME (APS)
No summary was provided in the NIHR record for this study.
Can the GH100 ACT be used to monitor heparin in Lupus anticoagulant?Evaluation of the Werfen GEM Hemochron 100 activated clotting time (ACT) point of care device in the therapeutic monitoring of unfractionated heparin in patients with Lupus anticoagulant whilst undergoing cardiopulmonary bypass.
Evaluates whether a prolonged aPTT due to lupus anticoagulant compromises laboratory-based unfractionated heparin monitoring during cardiopulmonary bypass, and tests the Werfen GEM Hemochron 100 point-of-care ACT device as an in-theatre alternative. Three groups: patients with a prolonged aPTT undergoing open-heart surgery on bypass, patients with a prolonged aPTT under Haematology Diagnostic clinic investigation (non-surgical), and a normal-coagulation-screen control group. Aim is to remove the turnaround delay of sending samples to an off-site laboratory and to establish whether point-of-care ACT tracks reliably against standard-care heparin monitoring in this population.