NCT06111638

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

A Phase 1/2/3 Open-label 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 Participants With Hemophilia A

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 55 (estimated)
• 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-H803 injection in severe 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.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
GENETIC	Single dose intravenous injection of BBM-H803	Single dose intravenous infusion of BBM-H803, an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene in liver. The dose of BBM-H803 will be calculated according to participant's weight.

Timeline

• Start date: 2024-01-03
• Primary completion: 2027-05-30
• Completion: 2031-05-30
• First posted: 2023-11-01
• Last updated: 2026-03-17

Locations

10 sites across 1 country: China