Actively Recruiting
A Clinical Study of AAV Vector Expressing Human Coagulation Factor FVIII Gene Therapy for Hemophilia A
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-02-21
8
Participants Needed
1
Research Sites
143 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 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 IU/dl). 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 1×10^13 vector genomes per kilogram. BBM 002 is designed to increase the body's 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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male subjects 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 protein products
- Documented bleeding events and/or infusions with factor VIII products in the last 12 weeks
- No history of hypersensitivity or anaphylaxis to factor VIII or intravenous immunoglobulin
- No factor VIII inhibitor detected (less than 0.6 Bethesda Units per ml) confirmed twice within 1-4 weeks
- Agree to use reliable barrier contraception from consent signing to 52 weeks post-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
- Presence of coagulation disorders other than Hemophilia A
- Use of systemic immunosuppressants other than glucocorticoids within 30 days prior to enrollment
- Vaccination within 30 days prior to screening
- Potential liver diseases such as portal hypertension, splenomegaly, hepatic encephalopathy, liver fibrosis stage 3 or higher, liver nodules or cysts, or elevated alpha-fetoprotein if clinically significant
- Planned major surgery during the 52-week study period
- Participation in prior gene therapy trials or investigational drug studies within 5 half-lives of the drug
- Alcohol or drug dependence or inability to abstain from alcohol during the study
- Any other major clinically significant disease or condition deemed unsuitable by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 52 weeks
Participants receive a single dose intravenous infusion of BBM 002, a gene therapy vector containing the human factor VIII gene.
Multiple visits for safety and efficacy monitoring during 52 weeks
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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