A Phase 1/2/3 Open-label 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
Overview
- Phase
- Phase 2
- Intervention
- Not specified
- Conditions
- Hemophilia B
- Sponsor
- Shanghai Belief-Delivery BioMed Co., Ltd
- Enrollment
- 32
- Locations
- 9
- Primary Endpoint
- Phase 1/2: The incidence of dose limiting toxicity (DLT) events
- Status
- Active, not recruiting
- Last Updated
- 10 months ago
Overview
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.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
Phase 1/2: The incidence of dose limiting toxicity (DLT) events
Time Frame: 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)
Time Frame: 10 weeks post-infusion
To assess the safety of BBM-H901 Injection by AEs and SAEs.
Phase 1/2: Changes in liver function
Time Frame: 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)
Time Frame: 52 weeks post-infusion
To assess ABR, including spontaneous bleeding and traumatic bleeding after administration.
Secondary Outcomes
- Phase 1/2: Changes in liver function(52 weeks post-infusion)
- Phase 1/2/3: Mean FIX Padua Activity Level(52 weeks post-infusion)
- Phase 1/2/3: Other FIX Protein Product Usage(52 weeks post-infusion)
- Phase 1/2/3: Target Joint Count(52 weeks post-infusion)
- Phase 1/2/3: Joint Bleeding Episodes(52 weeks post-infusion)
- Phase 1/2/3: Bleeding-Free Subjects(52 weeks post-infusion)
- Phase 1/2/3: Adverse Event Incidence(52 weeks post-infusion)
- Phase 1/2/3: FIX Inhibitor Incidence(52 weeks post-infusion)
- Phase 1/2/3: AAV Vector Shedding(52 weeks post-infusion)