Hemophilia A Single-Arm Study 2 - Healing Genes

Hemophilia A Single-Arm Study 2

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

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

Phase 3

Doctors at 72 research locations seek male patients with severe Hemophilia A (with Factor VIII Inhibitors) to trial a new product, Valoctocogene Roxaparvovec, an investigatory gene therapy. This therapy utilizes a virus to deliver a functional copy of the factor VIII (F8) gene to the liver, which may permanently provide sufficient blood coagulation and reduce the need for transfusions. This treatment has been studied in 2 prior trials, but the current study evaluates if the patients require less F8 infusions, if the liver cells are producing F8 successfully, and how the body responds to the study drug

The treatment requires a single intravenous injection of Valoctocogene Roxaparvovec and follow up will involve lab assessments and check-ins over the following year.


  • Be male and 18 years or older
  • Have congenital severe or moderately severe hemophilia A
  • Liver organ health meets clinical criteria


  1. Screening before the treatment
  2. A single infusion of the gene therapy drug will be administered
  3. Monitoring of the patient for a few hours after dosing, with periodic lab assessments required for 52 weeks.

Trials will take place at 68 locations across the United States. See the list of locations.
Contact: Trial Specialist  |  1-800-983-4587  |  [email protected]
BioMarin Pharmaceutical
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