Records containing: Hemophilia A
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CLINICAL_TRIALS study record update · 2026-03-17A Phase 1/2/3 Open-label Study to Evaluate the Safety, Tolerability and Efficacy of an Adeno-associated Viral Vector Containing an Expression Cassette of the Human Factor VIII Transgene (BBM-H803) Injection in Participants With Hemophilia A
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CLINICAL_TRIALS study record update · 2026-03-10A Phase 1 Single and Multiple Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Healthy Participants (Part A) and the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of SR604 in Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency (Part B)
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CLINICAL_TRIALS study record update · 2026-02-18Evaluation of Long-term Safety of ADYNOVI/ADYNOVATE (Antihaemophilic Factor [Recombinant] PEGylated, Rurioctocog Alfa Pegol) in Patients With Haemophilia A - An ADYNOVI/ADYNOVATE Post-Authorisation Safety Study (PASS)
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CLINICAL_TRIALS study record update · 2026-02-18An Open-label, Multicentre Study to Compare the Pharmacokinetics of Efanesoctocog Alfa Versus rFVIII Products, Damactocog Alfa Pegol or Turoctocog Alfa Pegol, After a Single Intravenous Dose of 50 IU/kg in a Fixed Sequence in Previously Treated Adults With Severe Haemophilia A.
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CLINICAL_TRIALS study record update · 2026-02-18aPCC and Emicizumab Safety Study in Congenital Hemophilia A Patients With Inhibitors (SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis)
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CLINICAL_TRIALS study record update · 2026-02-17Hemophilia A Research Program (HARP): An Observational Intergenerational Cohort Study of Hemophilia A and Factor VIII Immunogenicity
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CLINICAL_TRIALS study record update · 2026-02-12National Longitudinal Cohort of Hematological Diseases (NICHE)
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CLINICAL_TRIALS study record update · 2026-01-20Prospective, Observational Study of the Impact of Efanesoctocog Alfa (ALTUVIIIO®) on Goal Attainment and Physical Activity in People With Moderate or Severe Hemophilia A
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CLINICAL_TRIALS study record update · 2026-01-16Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis -STEP: SporTs Emicizumab Prophylaxis
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CLINICAL_TRIALS study record update · 2026-01-13Nuwiq Dosing and Outcomes In the ManagEment of Women/Girls With Haemophilia A Needing FVIII Treatment for Surgery - an International, Open-label, Non-controlled Study (NuDIMENSION)
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CLINICAL_TRIALS study record update · 2026-01-12A Long-Term Follow-Up Study in Subjects With Severe Hemophilia A Who Received BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in a Prior BioMarin Clinical Trial
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CLINICAL_TRIALS study record update · 2026-01-02Report of a Clinical-biological Case: "Long-term Anticoagulation in a Patient With Severe Hemophilia A
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CLINICAL_TRIALS study record update · 2025-12-09A Single-arm, Open-label, Multicenter Study Evaluating the Efficacy and Safety of GS1191-0445 Injection as a Single Dose in Chinese Subjects With Hemophilia A
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CLINICAL_TRIALS study record update · 2025-11-13Phase I Study Evaluating Safety and Feasibility of Hematopoietic Stem Cell Gene Transfer That Targets Factor VIII Delivery From Platelets for Patients With Hemophilia A
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CLINICAL_TRIALS study record update · 2025-11-12A Multicenter, Prospective, Open-label, Clinical Study to Assess the Effect of Using a New Risk Score Approach to Select the Most Appropriate Prophylaxis Regimen for Reaching a Favorable Outcome, When Hemophilia A Patients Switch From Standard Half-life Products to Damoctocog Alfa Pegol (Jivi)
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CLINICAL_TRIALS study record update · 2025-11-06AN OPEN-LABEL, NON-INVESTIGATIONAL PRODUCT, LEAD-IN STUDY TO EVALUATE AT LEAST 6 MONTHS OF PROSPECTIVE EFFICACY AND SAFETY DATA OF FACTOR IX OR FACTOR VIII PROPHYLAXIS REPLACEMENT THERAPY IN THE USUAL CARE SETTING OF MODERATELY SEVERE TO SEVERE ADULT HEMOPHILIA B SUBJECTS (FIX:C≤2%) WHO ARE NEGATIVE FOR nAb TO AAV VECTOR-SPARK100 AND MODERATELY SEVERE TO SEVERE HEMOPHILIA A ADULT SUBJECTS (FVIII:C≤1%) WHO ARE NEGATIVE FOR nAb TO AAV VECTOR SB-525 CAPSID (AAV6), PRIOR TO THE RESPECTIVE THERAPEUTIC PH 3 GENE THERAPY STUDIES (See Detailed Description Section for Official Protocol Title)
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CLINICAL_TRIALS study record update · 2025-10-23Drug Use Investigation of Kovaltry in Hemophilia A Patients
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CLINICAL_TRIALS study record update · 2025-10-02PROphylactic Efanesoctocog Alfa Therapy Evaluated for Critical Joint Health in Hemophilia A Treatment: The PROTECT-ALT Study
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CLINICAL_TRIALS study record update · 2025-10-02Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A
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CLINICAL_TRIALS study record update · 2025-09-30A PHASE 1/2, OPEN-LABEL, ADAPTIVE, DOSE-RANGING STUDY TO ASSESS THE SAFETY AND TOLERABILITY OF SB-525 (PF-07055480) (RECOMBINANT AAV2/6 HUMAN FACTOR 8 GENE THERAPY) IN ADULT SUBJECTS WITH SEVERE HEMOPHILIA A
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CLINICAL_TRIALS study record update · 2025-09-30An Open-label, Single-arm Treatment Study to Investigate the Safety and Tolerability of Switching From Emicizumab to Fitusiran Prophylaxis in Male Participants Aged ≥12 Years of Age With Severe Hemophilia A, With or Without Inhibitors
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CLINICAL_TRIALS study record update · 2025-09-24Effects of Emicizumab Treatment on the Musculoskeletal System in Children With Hemophilia A: A Prospective Study
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CLINICAL_TRIALS study record update · 2025-09-18A Phase 1, Single-Site, Open-Label Study to Assess Pharmacokinetics of Efanesoctocog Alfa (BIVV001), Standard Half-Life and Extended Half-Life FVIII After Each Single Intravenous Injection in a Fixed Sequence, in Previously Treated Adults With Severe Hemophilia A
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CLINICAL_TRIALS study record update · 2025-09-11A Global, Open-Label, Multicenter, Phase 1/2 Study of the Safety and Dose Escalation of BAX 888, an Adeno-Associated Virus Serotype 8 (AAV8) Vector Expressing B-Domain Deleted Factor VIII (BDD-FVIII) in Severe Hemophilia A Subjects Administered a Single Intravenous Infusion
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CLINICAL_TRIALS study record update · 2025-09-08Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A