Clinical Trial

Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug

Active, Not Recruiting Phase 2/3
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Summary
This is a multi-center, Phase 1/2/3, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H901 injection in Hemophilia B subjects with ≤2 International unit per deciliter (IU/dl) residual factor IX (FIX) levels. BBM-H901 is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene and raises circulating levels of endogenous FIX.
Protocol Amendment History 11 amendments
This ClinicalTrials.gov record has been amended 11 times since 2022-01-18; most recent amendment 2026-03-26.
Status change: Recruiting → Active, Not Recruiting 2023-08-09
Trial Details
NCT Number NCT05203679
Lead Sponsor Shanghai Xinzhi BioMed Co., Ltd.
Conditions Hemophilia B
Enrollment 32 participants
Start Date 2021-12-30
Primary Completion 2024-04-16 (estimated)
Study Completion 2028-06-30 (estimated)
Updated on ClinicalTrials.gov 2026-04-01