NCT05454774

A Study of FVIII Gene Therapy for Hemophilia A

A Clinical Study of AAV Vector Expressing Human Coagulation Factor FVIII Gene Therapy for Hemophilia A

• Status: Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 8 (estimated)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: Male
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a single-arm, open-label, clinical study to evaluate the safety, tolerability of BBM 002 injection in Hemophilia A subjects with residual factor VIII (FVIII) levels ≤2 International unit per deciliter (IU/dl) . BBM 002 injection is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII (hFVIII) transgene and raises circulating levels of endogenous FVIII.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
GENETIC	Single dose intravenous injection of BBM 002	Single dose intravenous infusion of BBM 002, an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII (hFVIII) transgene in liver. The dose of BBM 002 will be 1×10\^13 vg/ kg

Timeline

• Start date: 2022-07-19
• Primary completion: 2024-12-15
• Completion: 2027-09-15
• First posted: 2022-07-12
• Last updated: 2025-02-21

Locations

1 site across 1 country: China