Status:
COMPLETED
Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Hemophilia A
Eligibility:
MALE
18+ years
Phase:
PHASE3
Brief Summary
This Phase III clinical study will assess the impact of BMN 270 (compared to FVIII prophylaxis) on the number of bleeding episodes irrespective of exogenous FVIII replacement treatment in the efficacy...
Eligibility Criteria
Inclusion
- 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.
- 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 (EDs).
- No previous documented history of a detectable FVIII inhibitor, and results from a Bethesda assay or Bethesda assay with Nijmegen modification of less than 0.6 Bethesda Units (BU) on 2 consecutive occasions at least one week apart within the past 12 months.
Exclusion
- Detectable pre-existing antibodies to the adeno-associated virus 5 (AAV5) capsid.
- Any evidence of active infection or any immunosuppressive disorder, except for HIV infection
- Any evidence of active infection or any immunosuppressive disorder, including HIV infection (effective as of Protocol Amendment 3)
- Significant liver dysfunction.
- Prior liver biopsy showing significant fibrosis.
- Evidence of any bleeding disorder not related to hemophilia A.
- Platelet count of \< 100 x 10\^9/L.
- Creatinine ≥ 1.5 mg/dL.
- Liver cirrhosis of any etiology as assessed by liver ultrasound.
- Chronic or active hepatitis B.
- Active Hepatitis C.
- Active malignancy, except non-melanoma skin cancer.
- History of hepatic malignancy.
- History of arterial or venous thromboembolic events.
- Known inherited or acquired thrombophilia, including conditions associated with increased thromboembolic risk, such as atrial fibrillation.
Key Trial Info
Start Date :
December 19 2017
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
November 20 2024
Estimated Enrollment :
144 Patients enrolled
Trial Details
Trial ID
NCT03370913
Start Date
December 19 2017
End Date
November 20 2024
Last Update
March 25 2025
Active Locations (49)
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1
Los Angeles Orthopedic Hospital, Orthopedic Hemophilia Treatment Center
Los Angeles, California, United States, 90007-2664
2
UC Davis Hemophilia Treatment Center
Sacramento, California, United States, 95817
3
University of California San Diego, Hematology and Oncology, Hemophilia &Thrombosis Treatment Center
San Diego, California, United States, 92122
4
UCSF Medical Center
San Francisco, California, United States, 94143-0106