Brief Title
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
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
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.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Change of the Median Factor VIII (FVIII) Activity
Secondary Outcome
Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy
Condition
Hemophilia A
Intervention
Valoctocogene Roxaparvovec
Study Arms / Comparison Groups
Valoctocogene Roxaparvovec Open Label
Description: Single administration of valoctocogene roxaparvovec at a dose of 4E13 vg/kg
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
1
Start Date
March 14, 2018
Completion Date
May 2023
Primary Completion Date
May 22, 2019
Eligibility 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.
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Medical Director, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT03392974
Organization ID
BMN 270-302
Secondary IDs
2017-003573-34
Responsible Party
Sponsor
Study Sponsor
BioMarin Pharmaceutical
Study Sponsor
Medical Director, MD, Study Director, BioMarin Pharmaceutical
Verification Date
August 2022