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NCT03392974 | COMPLETED | Hemophilia A

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

BioMarin Pharmaceutical

Brief Summary:

This Phase III clinical study will assess the efficacy of BMN 270 defined as FVIII activity, during weeks 49-52 following intravenous infusion of BMN 270 and assess the impact of BMN 270 on usage of exogenous FVIII replacement therapy and the number of bleeding episodes from week 5 to week 52.

Condition or disease

Hemophilia A

Intervention/treatment

Valoctocogene Roxaparvovec

Phase

PHASE3

Study Type : INTERVENTIONAL
Estimated Enrollment : 1 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : 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 at a Dose of 4E13vg/kg in Hemophilia A Patients With Residual FVIII Levels ≤1IU/dL Receiving Prophylactic FVIII Infusions
Actual Study Start Date : 2018-03-14
Estimated Primary Completion Date : 2019-05-22
Estimated Study Completion Date : 2023-06-05

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 18 Years
Sexes Eligible for Study: MALE
Accepts Healthy Volunteers:
Criteria
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.
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

Location Details

NCT03392974

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How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

Not yet recruiting

United States, Pennsylvania

Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States, 15213-4306