Haemophilia: who works on it

One card per institution. Filter by target, by country, or show only institutions in EU-13 (Horizon Europe widening) countries.

• All targets
• CD38 1
• CFTR 1
• DMD 1
• G6PD 0
• GLP-1 0
• HTT 1
• NF1 1
• PPAR 0
• SMN1 1
• TNF 0

EU-13 is a filter, not a ranking: it only narrows the list.

24 of 471 institutions, ordered by recent publications and open trials. This is not a ranking of quality.

Terminated, withdrawn and suspended trials with the reason the sponsor gave in the registry, quoted word for word.

• Withdrawn

  SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

  NCT04563520· Emory University· Updated 6 Nov 2026

  “The study was withdrawn because the pharmaceutical sponsor withdrew support for the study. No participants were enrolled before termination.”Registry text, quoted unchanged. Source

• Terminated

  Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

  NCT03307980· Pfizer· Updated 5 Oct 2026

  “The study was terminated early due to Sponsor and participants may transition into a long-term follow-up study. The decision to terminate the trial was not based on any safety and/or efficacy concerns.”Registry text, quoted unchanged. Source

• Terminated

  Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX

  NCT04628871· Sangamo Therapeutics· Updated 13 Aug 2026

  “Sangamo declared Chapter 11 Bankruptcy on 23Jun2026 and terminated the study on 21Jul2026.”Registry text, quoted unchanged. Source

• Terminated

  Safety and Efficacy of FVIIa to Prevent Excessive Bleeding in Hemophilia Patients With Inhibitors Undergoing Major Surgery

  NCT05695391· Laboratoire français de Fractionnement et de Biotechnologies· Updated 31 Jul 2026

  “Feasibility considerations - extended timelines to complete recruitment”Registry text, quoted unchanged. Source

• Withdrawn

  Topical and Local TXA in Facelifts - A Randomized Controlled Double Blinded Study

  NCT06345833· University of Minnesota· Updated 23 Jul 2026

  “Lack of Participants”Registry text, quoted unchanged. Source

• Terminated

  Home Monitoring in Hemophilia A

  NCT06702579· Radboud University Medical Center· Updated 22 Jun 2026

  “The study was discontinued due to a lack of available personnel to complete the study”Registry text, quoted unchanged. Source

• Terminated

  BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

  NCT06611436· Be Biopharma· Updated 15 Jun 2026

  “The study was terminated due to a strategic business decision and not due to safety concerns”Registry text, quoted unchanged. Source

• Withdrawn

  POCUS: Hemostatic Potential and Joint Health in Patients With Severe Hemophilia A on Novel Replacement Therapies

  NCT04690322· University of Texas Southwestern Medical Center· Updated 7 Apr 2026

  “Due to COVID-19 pandemic, recruitment was temporarily put on hold. We are withdrawing this registration due to a recent amendment to the protocol that includes major changes to data elements. A new registration will be submitted for the new protocol.”Registry text, quoted unchanged. Source

• Terminated

  Goal Attainment and Physical Activity in People With Hemophilia A

  NCT06530030· Sanofi· Updated 20 Jan 2026

  “Early discontinuation based on sponsor decision not driven by any safety concerns.”Registry text, quoted unchanged. Source

• Withdrawn

  Patient Interview Study to Explore the Impact of Gene Therapy in Hemophilia A & B

  NCT06634836· Pfizer· Updated 29 Aug 2025

  “Business decision”Registry text, quoted unchanged. Source

Trials whose registry record says individual participant data (IPD) will be shared.

• SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis

  NCT04563520· Emory University· Source

• A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B

  NCT03861273· Pfizer· Source

• A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A

  NCT07416604· Hoffmann-La Roche· Source

• A Research Study Investigating Denecimig (Mim8) in Adults With Acquired Haemophilia A

  NCT07830732· Novo Nordisk A/S· Source

• A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B

  NCT07285460· Sanofi· Source

• A Study to Describe Patient REal-world FitusiRan ExpeRience

  NCT07895381· Sanofi· Source

• Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors

  NCT05145127· Pfizer· Source

• A Clinical Study to Evaluate the Effects of NXT007 Compared to Factor VIII Prophylaxis in Participants With Hemophilia A

  NCT07416526· Hoffmann-La Roche· Source

• A Non-Investigational Product, Multi-Center Cohort Study to Describe Long-Term Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec

  2025-523660-21-00· Pfizer Inc.· Source

• Recombinant Single-chain Factor VIII (rVIII-SingleChain) in Chinese Participants With Hemophilia A Previously Treated With FVIII Products

  NCT06738485· CSL Behring· Source

Find publications on this disease by a MeSH method term, for example a sequencing or imaging method.

• Single-Cell Analysis 1
• CRISPR-Cas Systems 6
• Gene Expression Profiling 1
• Sequence Analysis, RNA 1
• Whole Genome Sequencing 2
• Proteomics 1
• Flow Cytometry 1
• Immunohistochemistry 1
• Magnetic Resonance Imaging 6
• Machine Learning 3
• Deep Learning 2
• Cohort Studies 9

• Personalized prophylactic therapy optimization in hemophilia A using a hybrid PK-PD-TTE model and deep RL.

  J Pharmacokinet Pharmacodyn, Jun 2026

• Investigating blood cell images for enhanced hematologic disorder detection using multi-scale feature learning with a hybrid deep learning model.

  Sci Rep, Nov 2025

See which targets of this disease your abstract mentions, and go to the people working on them.