NCT04684940已完成1 期
A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Active or Prior Inhibitors
适应症
干预措施
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 10
- 试验地点
- 12
- 主要终点
- Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270.
研究概览
简要总结
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII. Part A of the study will involve subjects who have active inhibitors to FVIII, and Part B involving subjects with a prior history of inhibitors.
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 documented prior residual FVIII activity ≤ 1 IU/dL including, but not limited to, at the time of detected inhibitors, at the time of signing the informed consent.
- •History of a positive inhibitor result with the first positive result at least 12 month prior to Screening.
- •Part A: Demonstrated no immunological tolerance to exogenous FVIII. Part B: Demonstrated tolerance to exogenous FVIII and negative FVIII inhibitor screening titer < 0.6 BU.
- •Prophylactic or on-demand hemophilia therapy in the last 12 months. Bleeding, inhibitor & hemophilia therapy Hx over previous 12 months.
- •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.
- •Willing to abstain from consumption of alcohol for at least the first 52 weeks following BMN 270 infusion.
排除标准
- •Detectable pre-existing antibodies to the AAV5 capsid.
- •Any evidence of active infection or any immunosuppressive disorder; patients with HIV infection and undetectable viral load are not excluded.
- •Currently undergoing, or plan to receive during the study, immune tolerance induction therapy or prophylaxis with FVIII (Part A only).
- •Significant renal dysfunction or liver dysfunction, infection or history of hepatic malignancy.
- •Evidence of any bleeding disorder not related to hemophilia A.
研究组 & 干预措施
Valoctocogene roxaparvovec Open Label
Experimental
Single administration of valoctocogene roxaparvovec at a dose of 6E13 vg/kg in Active Inhibitor Population (Part A) and Prior Inhibitor Population (Part B).
干预措施: Valoctocogene roxaparvovec (Biological)
结局指标
主要结局
Number of participants with treatment-related adverse events, as assessed by Common Terminology Criteria for Adverse Events (CTCAE) v5.0 after administration of BMN 270.
时间窗: 60 months
次要结局
- Change of the median Factor VIII activity.(60 months)
- A change in Factor VIII inhibitor titer (Part A) after administration of BMN 270.(60 months)
- Absence of recurrence of Factor VIII inhibitors (Part B) after administration of BMN 270.(60 months)
- Change in the annualized utilization of hemophilia therapy after administration of BMN 270(60 months)
- Change in the annualized number of bleeding episodes requiring exogenous hemophilia therapy after administration of BMN 270.(60 months)
研究者
研究点 (12)
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