Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old

Recruiting Phase 1 Interventional Study
Hemophilia B
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
12 – 18
Sex
Male
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
9 (estimated)
Sponsor
Institute of Hematology & Blood Diseases Hospital, China · Other
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About This Trial
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 circ…
Trial Locations
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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 a…
Contacts

Feng Xue, MD

+862223909240

xuefeng@ihcams.ac.cn

CONTACT

Shuo Chen, BS

+862223909009

Chenshuo@ihcams.ac.cn

CONTACT