Brief Title
Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug
Official Title
A Study to Evaluate the Safety, Tolerability and Efficacy of an Adeno-associated Virus Vector Containing an Expression Cassette of the Human Factor IX Transgene (BBM-H901) Injection in Patients With Hemophilia B
Brief Summary
This is a multi-center, single-arm, open-label, single-dose treatment clinical study to evaluate the safety, tolerability and efficacy of BBM-H901 injection in Hemophilia B subjects with ≤2 International unit per deciliter (IU/dl) residual factor IX (FIX) levels. BBM-H901 is an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene and raises circulating levels of endogenous FIX.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Incidence of dose limiting toxicity (DLT) events.
Secondary Outcome
FIX activity
Condition
Hemophilia B
Intervention
Single dose intravenous injection of BBM-H901
Study Arms / Comparison Groups
Arm of BBM-H901
Description: 1×10^13 vg/kg, Single-dose treatment
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Genetic
Estimated Enrollment
12
Start Date
December 30, 2021
Completion Date
June 30, 2028
Primary Completion Date
June 30, 2024
Eligibility Criteria
Inclusion Criteria: 1. Males ≥ 18 years of age; 2. Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels; 3. Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products based on historical data from the subjects' records/histories; 4. Have had bleeding events and/or injected with FIX protein products (including recombination and plasma source) during the last 12 weeks documented in the subjects' medical records; 5. Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration; 6. Agree to use a reliable barrier contraception method from the beginning of signing the informed consent to 52 weeks after administration. Exclusion Criteria: 1. Being positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus-DNA (HBV-DNA). Being positive for hepatitis C virus antibody (HCV-Ab) or hepatitis C virus RNA (HCV-RNA). Subjects with medical history of hepatitis B or C can be regarded as negative only when 2 required samplings are conducted at least 3 months apart and both test results of indicators aforementioned are negative, i.e. subjects with natural clearance and anti-viral therapy clearance for hepatitis B or C are eligible; 2. Have potential liver diseases, such as previous diagnosis of portal hypertension, splenomegaly, hepatic encephalopathy or liver fibrosis (fibrosis stage ≥ 3); nodules or cysts were found by B ultrasound, or elevated alpha-fetoprotein was detected by laboratory tests. Subjects who are not eligible for the study if the abnormalities are clinically significant regarding to the medical judgement of the investigator; 3. HIV positive patients; 4. Have participated in a previous gene therapy research trial before screening, or in a clinical study with an investigational drug within 5 half-life of the investigational product, whichever is longer; 5. Have alcohol or drug dependence, or cannot stop drinking throughout the study; 6. Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation in the study.
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Lei Zhang, MD, 16602669908, [email protected]
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT05203679
Organization ID
BBM001-CLN1001
Responsible Party
Sponsor
Study Sponsor
Shanghai Belief-Delivery BioMed Co., Ltd
Study Sponsor
Lei Zhang, MD, Study Chair, Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Verification Date
January 2023