NCT03392974已完成3 期
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
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 1
- 试验地点
- 2
- 主要终点
- Change of the Median Factor VIII (FVIII) Activity
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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).
排除标准
- •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.
结局指标
主要结局
Change of the Median Factor VIII (FVIII) Activity
时间窗: Week 52
Change of the FVIII activity, as measured by chromogenic substrate assay, at Week 52 post-BMN 270 infusion.
次要结局
- Change in the Annualized Utilization (IU/kg) of Exogenous FVIII Replacement Therapy(Weeks 5 through Week 52)
- Change in the Annualized Number of Bleeding Episodes Requiring Exogenous FVIII Replacement Treatment(Weeks 5 though Week 52)
研究者
研究点 (2)
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