Actively Recruiting
A Clinical Study of AAV Vector Expressing Human Factor VIII Gene Therapy for Hemophilia A Evaluating Single-Dose Intravenous BBM 002
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-02-21
8
Participants Needed
1
Research Sites
269 weeks
Total Duration
On this page
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 carrying the human factor VIII (hFVIII) gene, in adult males with Hemophilia A who have very low levels of factor VIII (FVIII) in their blood. This early phase 1 clinical study focuses on participants with residual FVIII levels of 2 International units per deciliter (IU/dl) or less. Participants will receive a single intravenous dose of BBM 002 at 1 x 10^13 vector genomes per kilogram. This gene therapy is designed to increase the body's production of FVIII by delivering the hFVIII gene to the liver. The study involves only one treatment group receiving this infusion, and the effects will be monitored over time. During the 52-week study, participants will be closely observed for dose limiting toxicities within the first 10 weeks and for any treatment-emergent or serious adverse events throughout the entire year. Researchers will perform regular assessments including medical record reviews, monitoring of bleeding events, FVIII activity levels, and safety evaluations. Participants will also be required to use reliable contraception during the study period and comply with study procedures to help ensure accurate results and safety.
CONDITIONS
Official Title
A Study of FVIII Gene Therapy for Hemophilia A
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male aged 18 years or older
- Diagnosed with Hemophilia A with endogenous factor VIII activity levels of 2 IU/dL or less
- At least 150 prior exposure days to recombinant and/or plasma-derived factor VIII products
- Documented bleeding events and/or factor VIII infusions in the past 12 weeks
- No history of hypersensitivity or anaphylaxis related to factor VIII or intravenous immunoglobulin
- No factor VIII inhibitor detected in two consecutive tests or prior medical history
- Agree to use reliable barrier contraception from informed consent signing to 52 weeks after BBM 002 infusion
- Good compliance and willingness to participate in gene therapy clinical trials
You will not qualify if you...
- Positive for hepatitis B surface antigen or hepatitis B virus DNA
- Positive for hepatitis C virus antibody or RNA
- Currently receiving antiviral therapy for hepatitis B or C
- Other coagulation disorders besides Hemophilia A
- Use of systemic immunosuppressants other than glucocorticoids within 30 days before enrollment
- Vaccination within 30 days prior to screening
- Presence of significant liver disease or abnormalities
- Planned major surgery during the 52-week study period
- Previous participation in gene therapy trials or investigational drug studies within 5 half-lives
- Alcohol or drug dependence or inability to abstain during the study
- Any other major clinically significant disease or condition deemed unsuitable by investigators
AI-Screening
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Trial Site Locations
Total: 1 location
1
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, China, 300020
Actively Recruiting
Research Team
L
Lei Zhang, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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