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

Hemophilia B Clinical Trial

Official title:
A 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

Clinical Trial Summary

This is a multi-center, 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05203679
Study type Interventional
Source Shanghai Belief-Delivery BioMed Co., Ltd
Contact
Status Active, not recruiting
Phase Phase 3
Start date December 30, 2021
Completion date June 30, 2028
View Details
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