A Phase 1/2 Safety, Tolerability, and Efficacy Study of BMN 270, an AdenoAssociated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients with Active or Prior Inhibitors -

BIOMARIN PHARMACEUTICAL INC.0 个研究点目标入组 20 人开始时间: 2021年6月7日最近更新: 2021年12月6日

研究概览

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•1. 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.
•2. History of a positive inhibitor result with the first positive result in the last 12 months. Part A: Positive FVIII inhibitor titer = 0.6 BU. Part B: Negative FVIII inhibitor screening titer < 0.6 BU.
•3. Part A: Emicizumab prophylaxis at least 6 months prior to screening; Part B: FVIII replacement prophylaxis for 12 months. Bleeding, inhibitor & hemophilia therapy Hx over previous 12 months.
•4. 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.
•5. Willing to abstain from consumption of alcohol for at least the first 52 weeks following BMN 270 infusion.
•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 17
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 3

•1. Detectable pre-existing antibodies to the AAV5 capsid.
•2. Any evidence of active infection or any immunosuppressive disorder, including HIV infection.
•3. Currently undergoing, or plan to receive during the study, immune tolerance induction therapy or prophylaxis with FVIII (Part A only).
•4. Significant renal dysfunction or liver dysfunction, infection or history of hepatic malignancy.
•5. Evidence of any bleeding disorder not related to hemophilia A.

发起方