Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug
- Conditions
- Hemophilia B
- Interventions
- Genetic: Single dose intravenous injection of BBM-H901
- Registration Number
- NCT05203679
- Lead Sponsor
- Shanghai Belief-Delivery BioMed Co., Ltd
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- Male
- Target Recruitment
- 32
- Males ≥ 18 years of age;
- Have hemophilia B with ≤2 IU/dL (≤2 %) endogenous FIX activity levels;
- 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;
- 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;
- Have no prior history of hypersensitivity or anaphylaxis associated with any FIX or IV immunoglobulin administration;
- Agree to use a reliable barrier contraception method from the beginning of signing the informed consent to 52 weeks after administration.
- 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;
- 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;
- HIV positive patients;
- 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;
- Have alcohol or drug dependence, or cannot stop drinking throughout the study;
- Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation in the study.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Arm of BBM-H901 Single dose intravenous injection of BBM-H901 1×10\^13 vg/kg, Single-dose treatment
- Primary Outcome Measures
Name Time Method Annualized bleeding rate (ABR) 52 weeks post-infusion To assess ABR, including spontaneous bleeding and traumatic bleeding after administration.
- Secondary Outcome Measures
Name Time Method Mean FIX Padua Activity Level 52 weeks post-infusion Measurement of mean FIX Padua activity levels over the 52-week period following BBM-H901 injection.
Other FIX Protein Product Usage 52 weeks post-infusion Number and total volume of infusions of exogenous FIX protein products (recombinant or plasma-derived) administered within 52 weeks post-BBM-H901 injection.
Target Joint Count 52 weeks post-infusion Number of target joints recorded within 52 weeks post-BBM-H901 injection.
Joint Bleeding Episodes 52 weeks post-infusion Total number of joint bleeding events occurring within 52 weeks post-BBM-H901 injection.
Bleeding-Free Subjects 52 weeks post-infusion Proportion of subjects experiencing no bleeding events within 52 weeks post-BBM-H901 injection.
Adverse Event Incidence 52 weeks post-infusion Incidence of adverse events (AEs) and serious adverse events (SAEs) within 52 weeks post-BBM-H901 injection.
FIX Inhibitor Incidence 52 weeks post-infusion Incidence of FIX inhibitors measured by Bethesda or Nijmegen-Bethesda assays within 52 weeks post-BBM-H901 injection.
AAV Vector Shedding 52 weeks post-infusion Changes in AAV vector shedding in plasma, urine, semen, saliva, and PBMCs within 52 weeks post-BBM-H901 injection.
Trial Locations
- Locations (9)
The Second People's Hospital of Shenzhen
🇨🇳Shenzhen, Guangdong, China
Peking Union Medical College Hospital
🇨🇳Beijing, Beijing, China
Anhui Provincial Hospital
🇨🇳Hefei, Anhui, China
North China University of Science and Technology Affiliated Hospital
🇨🇳Tangshan, Hebei, China
Henan Cancer Hospital
🇨🇳Zhengzhou, Henan, China
Nanfang Hospital Southern Medical University
🇨🇳Guangzhou, Guangdong, China
The Second Hospital of Shanxi Medical University
🇨🇳Taiyuan, Shanxi, China
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
🇨🇳Tianjin, Tianjin, China
The second Affiliated Hospital of Kunming Medical University
🇨🇳Kunming, Yunnan, China