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Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs

Hemophilia A Clinical Trial

Official title:
A Study to Evaluate the Safety, Tolerability and Efficacy of an Adeno-associated Viral Vector Containing an Expression Cassette of the Human Factor VIII Transgene (BBM-H803) Injection in Patients With Hemophilia A

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-H803 injection in heavy Hemophilia A subjects. BBM-H803 is an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene and raises circulating levels of endogenous FVIII.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06111638
Study type Interventional
Source Shanghai Belief-Delivery BioMed Co., Ltd
Contact Li Chen, Master
Phone 16602669908
Email ra@beliefbiomed.com
Status Recruiting
Phase Phase 1/Phase 2
Start date October 31, 2023
Completion date June 30, 2030
View Details
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