Hemophilia A Single-Arm Study 1 - Healing Genes

Hemophilia A Single-Arm Study 1

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Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg (BMN270-302)

Phase 3 Study To Evaluate Efficacy/Safety of Valoctocogene Roxaparvovec an AAV Vector-Mediated Gene Transfer of hFVIII at a Dose of 4E13vg/kg in Hemophilia A Patients With Residual FVIII Levels ≤1IU/dL Receiving Prophylactic FVIII Infusions

Phase 3

Doctors at 68 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 will see if and how the body responds to the study drug – for example, whether liver cells become inflamed or whether the body makes antibodies (something the immune system makes to protect itself against things like bacteria and viruses) against the vector or the new Factor VIII gene.

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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