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Hemophilia clinical trials at UCSF

13 in progress, 3 open to eligible people

Showing trials for
  • 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

    open to eligible males ages 18-65

    This study will evaluate the efficacy and safety of PF-06838435 (a gene therapy drug) in adult male participants with moderately severe to severe hemophilia B (participants that have a Factor IX circulating activity of 2% or less). The gene therapy is designed to introduce genetic material into cells to compensate for missing or non-functioning Factor IX. Eligible study participants will have completed a minimum 6 months of routine Factor IX prophylaxis therapy during the lead in study (C0371004). Participants will be dosed once (intravenously) and will be evaluated over the course of 6 years. The main objectives of the study are to compare the annualized bleeding rate [ABR] of the gene therapy to routine prophylaxis from the lead-in study and to evaluate the impact that it may have on participant's Factor IX circulating activity [FIX:C].

    San Francisco, California and other locations

  • Six Month lead-in Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

    open to eligible males ages 18-64

    To establish a minimum of 6 months of prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study. To establish a minimum of 6 months of prospective efficacy data of current FVIII prophylaxis replacement therapy in the usual care setting of hemophilia A subjects, who are negative for nAb to SB-525 capsid (AAV6), prior to the Phase 3 gene therapy study. The enrollment for hemophilia A participants is completed. At this time participants are only being enrolled for hemophilia B cohort.

    San Francisco, California and other locations

  • Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

    open to eligible males ages 18-64

    C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants with moderately severe or severe hemophilia A (FVIII:C≤1%) for the study duration of 5 years. The study will enroll eligible participants who have been followed on routine prophylaxis with FVIII products in the Lead-In study C0371004.

    San Francisco, California and other locations

  • A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

    Sorry, in progress, not accepting new patients

    The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity after dosing with SB-525 (PF-07055480)

    San Francisco, California and other locations

  • ATHN 11: Liver Transplantation Outcomes Study

    Sorry, not currently recruiting here

    This study is designed as a multi-center, observational cohort study of participants with hemophilia A and B who have and have not undergone liver transplantation. Participants will be asked to complete health related quality of life questionnaires and provide medical history.

    San Francisco, California and other locations

  • ATHN 7: Hemophilia Natural History Study

    Sorry, in progress, not accepting new patients

    This is a real-world study of the safety of the treatments used for people with hemophilia. The study will follow people with hemophilia A or B from across the country for about 4 years as they receive treatment. The hemophilia treatment center (HTC) physician and participant will decide on the FDA-approved treatment to be used which may include non-factor products, bypassing agents, or clotting factor replacement products. The goal of this research is to study the use of hemophilia treatment products and their outcomes.

    San Francisco, California and other locations

  • ATHN 8: Previously Untreated Patients (PUPs) Matter Study

    Sorry, not currently recruiting here

    This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs). Participants will be followed as they receive their first 50 exposure days (ED) to clotting factor replacement product, both prospectively and retrospectively. The data collected on evolving treatment practices will define the incidence and risk factors for inhibitor development during the high risk period of first 50 ED and improve the outcomes of this vulnerable population.

    San Francisco, California and other locations

  • HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

    Sorry, in progress, not accepting new patients

    This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061). AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The pharmaceutical form of AMT-061 is a solution for intravenous infusion administered at a dose of 2 x 1013 gc/kg.

    San Francisco, California and other locations

  • Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants

    Sorry, in progress, not accepting new patients

    The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe hemophilia A receiving prophylactic FVIII replacement therapy as standard of care. The data collected from this study may assist in providing baseline information for comparison to the Spark's investigational hemophilia A gene therapy in future Phase 3 studies.

    San Francisco, California and other locations

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

    Sorry, accepting new patients by invitation only

    Long-term follow-up of subjects who received SB-318, SB-913, or SB-FIX in a previous trial and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 10 years following exposure to SB-318, SB-913, or SB-FIX.

    Oakland, California and other locations

  • Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis

    Sorry, not yet accepting patients

    Hemophilia A (HA) is a genetic bleeding disorder resulting from a deficiency or absence of factor VIII (FVIII), which is necessary in the clotting process. This disorder occurs mostly in males and in severe cases causes frequent bleeding episodes in joints and muscles which can lead to progressive damage that affects mobility and quality of life. Prophylactic FVIII administered intravenously every other day has been the standard of care treatment for HA for the past few decades. Sports and physical activity are generally encouraged in patients with hemophilia on appropriate prophylactic treatment to increase strength, prevent or decrease obesity, accrue and maintain bone density and encourage normal socialization. To ensure safety with participation in sports in persons with hemophilia A (PWHA), timing of FVIII administration is often adjusted to maximize FVIII at the time of sports. The exact factor level that is needed to safely participate in sports and minimize bleeding risk is not yet known. Based on clinical practice, infusion of FVIII to near the lower limit of normal right before participation in sports generally works to prevent bleeding. The study is looking at how well the newly approved medication Emicizumab works compared to Factor VIII to prevent bleeding in patients with Hemophilia A who play sports. The study will enroll children and adolescents who are already on Emicizumab or Factor VIII who are currently playing sports.

    San Francisco, California and other locations

  • Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

    Sorry, in progress, not accepting new patients

    This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week 5 to last visit by data cutoff.

    San Francisco, California and other locations

  • Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders

    Sorry, not currently recruiting here

    The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.

    San Francisco, California and other locations

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