Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
Trial Parameters
Brief Summary
This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual FIX levels and aged 12-18 years old. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.
Eligibility Criteria
Inclusion Criteria: 1. Subjects and statutory guardian must be able to understand the purpose and risks of the study and provide signed and dated informed consent; 2. Be male and 12≤ age \<18 years of age, body wight ≥ 50kg; 3. Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels as documented by a certified clinical laboratory at the time of screening. If the screening result is \>2% due to insufficient washout from FIX protein product, then the severity of hemophilia B may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤2% FIX coagulant activity (FIX:C) ; 4. Had had ≥75 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products based on historical data from the subject's record/history; 5. With ≤ 1:4 neutralizing antibodies and ≤1:200 binding antibodies against BBM-H901 capsid; 6. Subjects with bleeding episode and/ or FIX agents infusion events within 12 weeks prior to screening; 7. Have