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 in Hemophilia A Patients with Residual FVIII Levels = 1 IU/dL Receiving Prophylactic FVIII Infusions

BioMarin Pharmaceutical Inc.0 个研究点目标入组 134 人开始时间: 2017年11月20日最近更新: 2021年10月8日

研究概览

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•- 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.
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 115
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 15

•- 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.
•- 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.

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