For more information on these or any other studies, please contact either Manny Mangilit or Dr. Quon at (213) 742-1402
- The ATHNdataset, a limited data set related to Bleeding and Clotting Disorders (Open to all ages/patients with bleeding disorders)
- CDC Public Health Surveillance for Bleeding Disorders – Registry for Bleeding Disorders Surveillance (Open to all ages/patients with bleeding disorders)
- A Multicenter Study of Eradication of Hepatitis C Virus (HCV) Infection in People with Hemophilia and Bleeding Disorders (ATHN 6) (Open to patients with bleeding disorders with hepatitis C, recently treated or untreated looking to be treated)
- A Natural History Cohort Study of the Safety, Effectiveness, and Practice of Treatment for People with Hemophilia (ATHN 7) (Open to all ages/All patients with Hemophilia A & B)
- Hemophilia B patients all ages: Musculoskeletal Ultrasound for the Joint Assessment of People with Bleeding Disorders (Open to adult patient with hemophilia B)
- An Observational Study of Postoperative Deep Venous Thrombosis (DVT) in Hemophilics Undergoing Major Orthopedic Surgery (Open to hemophilia A and B patients undergoing surgery)
- Biomarin: A Prospective Non-Interventional Study of Bleeding Episodes, Factor VIII Infusions, and Patient-Reported Outcomes in Individuals with Severe Hemophilia A (Open to only severe hemophilia A patients)
- Biomarin: A Phase 3 Open-Label, Single-Arm Study To Evaluate The Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector–Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Residual FVIII Levels ≤ 1 IU/dL Receiving Prophylactic FVIII Infusions (Open to only severe hemophilia A patients)
- Biomarin: Global Prospective Laboratory Study for Adenovirus-Associated Virus (AAV) Seroprevalence in Hemophilia A Patients with Residual Factor VIII Levels ≤2 IU/dL Who Were Previously Treated with Factor VIII Concentrates (Open to only hemophilia A patients)
- Baxalta/Takeda: A Global Epidemiologic Study to Determine the Prevalence of Neutralizing Antibodies and Related Adaptive Immune Responses to Adeno-Associated Virus (AAV) in Adults with Hemophilia (Open to only hemophilia A patients)
- Baxalta/Takeda: A 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 (Open to only severe hemophilia A patients)
- UniQure: Phase III, open-label, single-dose, multi-center multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately severe hemophilia B (Open to only severe adult hemophilia B patients)