Brief Title
Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
Official Title
A Phase 3b, Single Arm, Open-Label Study to Evaluate the Efficacy and Safety of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII, With Prophylactic Corticosteroids in Hemophilia A Patients
Brief Summary
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Change in median FVIII activity as measured by chromogenic substrate assay, after administration of BMN 270 with prophylactic corticosteroids
Secondary Outcome
Change in the annualized utilization (IU/kg) of exogenous FVIII replacement therapy or emicizumab for subjects receiving FVIII or emicizumab prophylaxis respectively after administration of BMN 270 with prophylactic corticosteroids
Condition
Hemophilia A
Intervention
valoctocogene roxaparvovec
Study Arms / Comparison Groups
valoctocogene roxaparvovec
Description: Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg with prophylactic corticosteroids
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
20
Start Date
November 10, 2020
Completion Date
January 2027
Primary Completion Date
January 2023
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, at the time of signing the informed consent. 2. Must have been on prophylactic hemophilia 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 (EDs). 4. No previous documented history of a detectable FVIII inhibitor, <0.6 Bethesda Units (BU). 5. Sexually active participants must agree to use an acceptable method of effective contraception. Participants must agree to contraception use for at least 12 weeks post-infusion. Exclusion Criteria: 1. Detectable pre-existing antibodies to the AAV5 capsid. Up to 25% of subjects may have detectable pre-existing AAV5 capsid antibodies, so long as the titer level is below the minimum required dilution (< 20). 2. Any evidence of active infection or any immunosuppressive disorder; patients with HIV infection and undetectable viral load are not excluded. 3. Significant renal dysfunction or liver dysfunction, infection or history of hepatic malignancy. 4. Evidence of any bleeding disorder not related to hemophilia A.
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Medical Monitor, MD, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT04323098
Organization ID
BMN 270-303
Secondary IDs
2018-004616-21
Responsible Party
Sponsor
Study Sponsor
BioMarin Pharmaceutical
Study Sponsor
Medical Monitor, MD, Study Director, BioMarin Pharmaceutical
Verification Date
February 2022