Status:
COMPLETED
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Hemophilia A
Eligibility:
MALE
18+ years
Phase:
PHASE3
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 e...
Eligibility Criteria
Inclusion
- Males ≥ 18 years of age with hemophilia A and residual FVIII levels ≤ 1 IU/dL as evidenced by medical history.
- Must have been on prophylactic FVIII replacement therapy for at least 12 months prior to study entry.
- Treated/exposed to FVIII concentrates or cryoprecipitate for a minimum of 150 exposure days.
- No previous documented history of a detectable FVIII inhibitor of less than 0.6 Bethesda Units (BU).
Exclusion
- Detectable pre-existing antibodies to the AAV5 capsid.
- Any evidence of active infection or any immunosuppressive disorder, including HIV infection.
- Significant liver dysfunction, prior liver biopsy showing significant fibrosis, liver cirrhosis of any etiology or history of hepatic malignancy.
- Evidence of any bleeding disorder not related to hemophilia A.
- Active Hepatitis C.
- Prior treatment with any vector/gene transfer agent.
Key Trial Info
Start Date :
March 14 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 5 2023
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT03392974
Start Date
March 14 2018
End Date
June 5 2023
Last Update
October 3 2023
Active Locations (1)
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1
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States, 15213-4306