Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug
- Conditions
- Hemophilia B
- Registration Number
- NCT05203679
- Lead Sponsor
- Shanghai Belief-Delivery BioMed Co., Ltd
- Brief Summary
This is a multi-center, Phase 1/2/3, 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
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Primary Outcome Measures
Name Time Method Phase 1/2: The incidence of dose limiting toxicity (DLT) events 10 weeks post-infusion To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) in DLT observation period after BBM-H901 injection infusion.
Phase 1/2: The incidence of adverse events (AEs) and serious adverse events (SAEs) 10 weeks post-infusion To assess the safety of BBM-H901 Injection by AEs and SAEs.
Phase 1/2: Changes in liver function 10 weeks post-infusion To assess changes in liver function before and after treatment, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST).
Phase 3: 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 Phase 1/2: Changes in liver function 52 weeks post-infusion To assess changes in liver function before and after treatment, including alanine aminotransferase (ALT) and aspartate aminotransferase (AST).
Phase 1/2/3: 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.
Phase 1/2/3: 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.
Phase 1/2/3: Target Joint Count 52 weeks post-infusion Number of target joints recorded within 52 weeks post-BBM-H901 injection.
Phase 1/2/3: Joint Bleeding Episodes 52 weeks post-infusion Total number of joint bleeding events occurring within 52 weeks post-BBM-H901 injection.
Phase 1/2/3: Bleeding-Free Subjects 52 weeks post-infusion Proportion of subjects experiencing no bleeding events within 52 weeks post-BBM-H901 injection.
Phase 1/2/3: Adverse Event Incidence 52 weeks post-infusion Incidence of adverse events (AEs) and serious adverse events (SAEs) within 52 weeks post-BBM-H901 injection.
Phase 1/2/3: 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.
Phase 1/2/3: 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)
Anhui Provincial Hospital
🇨🇳Hefei, Anhui, China
Peking Union Medical College Hospital
🇨🇳Beijing, Beijing, China
Nanfang Hospital Southern Medical University
🇨🇳Guangzhou, Guangdong, China
The Second People's Hospital of Shenzhen
🇨🇳Shenzhen, Guangdong, China
North China University of Science and Technology Affiliated Hospital
🇨🇳Tangshan, Hebei, China
Henan Cancer Hospital
🇨🇳Zhengzhou, Henan, 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
Anhui Provincial Hospital🇨🇳Hefei, Anhui, China