Actively Recruiting
Study of Single-Dose BBM 002 Gene Therapy for Hemophilia A in Adult Men
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-02-21
8
Participants Needed
1
Research Sites
143 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and tolerability of BBM 002 injection, a gene therapy using an adeno-associated virus AAV vector that carries the human factor VIII gene. This study focuses on males with Hemophilia A who have very low levels of factor VIII less than or equal to 2 IUdl. The trial is an early phase 1, single-arm, open-label study designed to assess this investigational genetic treatment. Participants will receive a single intravenous dose of BBM 002 at 11013 vector genomes per kilogram. BBM 002 is designed to increase the bodys production of factor VIII, which is important for blood clotting. This one-time infusion is the only treatment administration in the study. During the study, participants will be monitored for safety, including dose limiting toxicities within 10 weeks and treatment-emergent and serious adverse events over 52 weeks. Researchers will evaluate participants bleeding history, factor VIII levels, and adverse reactions. The total follow-up duration is at least one year to track safety and response to the gene therapy.
CONDITIONS
Brief Title
A Study of FVIII Gene Therapy for Hemophilia A
Research Team
L
Lei Zhang, MD
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