This Phase 3 study assesses the efficacy of BMN 270 (valoctocogene roxaparvovec) gene therapy, defined as FVIII activity during weeks 49-52 after intravenous infusion. It also evaluates the impact on use of exogenous FVIII replacement therapy and the number of bleeding episodes from week 5 to week 52. Participants are males aged 18 or older with hemophilia A and residual FVIII levels of 1 IU/dL or less by medical history, who had been on prophylactic FVIII replacement therapy for at least 12 months before study entry.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: 1. Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL as evidenced by medical history. 2. Must have been on prophylactic FVIII replacement therapy for at least 12 months prior to study entry. 3. Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days. 4. No previous documented history of a detectable FVIII inhibitor of less than 0.6 Bethesda Units (BU). Exclusion Criteria: 1. Detectable pre-existing antibodies to the AAV5 capsid. 2. Any evidence of active infection or any immunosuppressive disorder, including HIV infection. 3. Significant liver dysfunction, prior liver biopsy showing significant fibrosis, liver cirrhosis of any etiology or history of hepatic malignancy. 4. Evidence of any bleeding disorder not related to hemophilia A. 5. Active Hepatitis C. 6. Prior treatment with any vector/gene transfer agent.