Actively Recruiting
Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-02-21
9
Participants Needed
1
Research Sites
658 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a Phase 1, open- label, non- randomized, uncontrolled, single dose pilot study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of BBM-H901 in hemophilia B subjects with ≤2IU/dl residual FIX levels and aged 12-18 years old. BBM-H901 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.
CONDITIONS
Official Title
Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male participants aged 12 to less than 18 years
- Body weight of at least 50 kg
- Diagnosed with hemophilia B with endogenous factor IX activity levels of 2 IU/dL or less confirmed by certified laboratory
- At least 75 prior exposure days to any recombinant or plasma-derived factor IX products
- Neutralizing antibodies against BBM-H901 capsid at 1:4 or less and binding antibodies at 1:200 or less
- Experienced bleeding episodes or factor IX infusions within 12 weeks before screening
- No history of hypersensitivity or anaphylaxis to factor IX or intravenous immunoglobulin
- No measurable factor IX inhibitor or no history of factor IX inhibitor and no clinical signs of reduced response to factor IX
- Acceptable lab values: hemoglobin 11 g/dL or higher, platelets 100,000 cells/µL or higher, AST and ALT less than or equal to 1.5 times upper normal limit, bilirubin less than or equal to 1.5 times upper normal limit, and estimated glomerular filtration rate 60 ml/min or higher
- For sexually mature subjects, agreement to use reliable barrier contraception until 52 weeks
- Good compliance with study visit schedule and diary completion
You will not qualify if you...
- Positive for hepatitis B surface antigen antibody or HBV-DNA, or hepatitis C antibody or HCV-RNA
- Currently receiving antiviral therapy for hepatitis B or C
- Having coagulation disorders other than hemophilia B
- Received immunosuppressive therapy other than steroids within 30 days before screening
- Received vaccine within 30 days before screening or planned vaccination during the study period up to 52 weeks
- Significant underlying liver disease or other liver conditions unsuitable for gene therapy
- Planned surgery within 52 weeks after gene therapy
- History of chronic infection or high risk of infection deemed unacceptable by investigator
- Participation in gene therapy trials within the past 52 weeks or investigational drug trials within 12 weeks
- Use of herbs affecting liver function within 4 weeks before screening
- History of fatal bleeding episodes such as intracranial hemorrhage
- Any other major clinically significant disease or condition making participation unsuitable as judged by the investigator
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Institute of haematology and Blood diseases hospital
Tianjin, Tianjin Municipality, China, 300020
Actively Recruiting
Research Team
F
Feng Xue, MD
CONTACT
S
Shuo Chen, BS
CONTACT
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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