• Hemophilia A Gene Therapy Clinical Trial

    Gene Therapy

    Phone: 617-632-5064  

    Gene-transfer, open-label, dose escalation study of SPK-8011 [adeno-associated viral vector with B-domain deleted human factor VIII gene] in individuals with hemophilia A

    Hemophilia A is an X-linked recessive genetic bleeding disorder caused by mutations in the factor 8 (F8) gene. FVIII is produced in liver endothelial cells and is critical for fibrin clot formation. Severe hemophilia A is characterized by frequent, spontaneous internal bleeding that can lead to chronic joint damage, intracranial hemorrhage, and even death. Current treatment for hemophilia A is based on replacement of the deficient FVIII with IV injections of recombinant FVIII clotting protein prophylactically or as needed to treat bleeding episodes.

    This gene therapy clinical trial for hemophilia A evaluates whether the AAV vector carrying a F8 gene can increase FVIII levels in patients with moderate to severe hemophilia A. Specifically, the study will evaluate the safety and efficacy of the adeno-associated virus (AAV) to deliver the human F8 gene, the healthy gene necessary to make FVIII, to the liver. This study will determine if SPK-8011 can produce clinically meaningful FVIII levels in patients with moderately/severe or severe hemophilia A.

    Eligibility criteria:

    • Male 18 years of age or older
    • Moderate/severe or severe hemophilia A (baseline FVIII activity <1% of normal or documented history of FVIII activity <1%)
    • Have had > 150 prior exposure days to any recombinant and/or plasma-derived FVIII concentrates or cryoprecipitates
    • Have no prior history of hypersensitivity or anaphylaxis associated with FVIII or IV administration
    • Have no measurable inhibitor against FVIII, no prior history of FVIII inhibitor and no clinical signs or symptoms of decrease response to FVIII
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