NCT05203679

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

A Phase 1/2/3 Open-label Study to Evaluate the Safety, Tolerability and Efficacy of an Adeno-associated Virus Vector Containing an Expression Cassette of the Human Factor IX Transgene (BBM-H901) Injection in Patients With Hemophilia B

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 32 (actual)
• Sponsor: Shanghai Xinzhi BioMed Co., Ltd. · Industry
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia B

Interventions

Type	Name	Description
GENETIC	Single dose intravenous injection of BBM-H901	Single dose intravenous infusion of BBM-H901, an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene in liver.

Timeline

• Start date: 2021-12-30
• Primary completion: 2024-04-16
• Completion: 2028-06-30
• First posted: 2022-01-24
• Last updated: 2026-04-01

Locations

9 sites across 1 country: China