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A Study of FVIII Gene Therapy for Hemophilia A
Sponsor: Institute of Hematology & Blood Diseases Hospital, China
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.
Official title: A Clinical Study of AAV Vector Expressing Human Coagulation Factor FVIII Gene Therapy for Hemophilia A
Key Details
Gender
MALE
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
8
Start Date
2022-07-19
Completion Date
2027-09-15
Last Updated
2025-02-21
Healthy Volunteers
No
Conditions
Interventions
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
Locations (1)
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, China