• Hemophilia Gene Therapy Clinical Trial

    Gene Therapy

    Phone: 617-632-5064  

    Safety and Dose Finding Study of DTX101 (AAVrh10FIX) in Adults with Moderate/Severe to Severe Hemophilia B

    Hemophilia B is an X-linked recessive genetic bleeding disorder caused by mutations in the factor IX (FIX) gene. FIX is produced in the liver and is critical for fibrin clot formation. Hemophilia B is characterized by frequent, spontaneous internal bleeding that can lead to chronic joint damage, intracranial hemorrhage, and even death. Current treatment for hemophilia B is based on replacement of the deficient FIX with IV injections of recombinant FIX protein prophylactically or as needed to treat bleeding episodes.

    Our gene therapy clinical trial for hemophilia B evaluates whether the AAV vector carrying a FIX gene can increase FIX levels in patients with moderate to severe hemophilia B. Specifically, the study will evaluate the safety and efficacy of the adeno-associated virus (AAV) to deliver the human factor IX (hFIX) gene, the healthy gene necessary to make FIX, to the liver where FIX is normally produced. This study will determine if AAVrh10 can produce clinically meaningful FIX levels in patients with moderately/severe or severe hemophilia B.

    Eligibility criteria:

    • Male 18 years of age or older
    • Moderate/severe or severe hemophilia B (baseline FIX activity ≤2% of normal or documented history of FIX activity ≤2%)
    • At least 3 bleeding episodes per year that require on-demand treatment with FIX OR are treated with a prophylactic regimen of FIX
    • No documented history of inhibitors (neutralizing antibodies) to exogenous FIX
    • No known allergic reaction to exogenous FIX
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