Status:
ACTIVE_NOT_RECRUITING
Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug
Lead Sponsor:
Shanghai Xinzhi BioMed Co., Ltd.
Conditions:
Hemophilia B
Eligibility:
MALE
18+ years
Phase:
PHASE2
PHASE3
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 ...
Eligibility Criteria
Inclusion
- Inclusion Criteria of Phase 1/2/3:
- 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.
- Exclusion Criteria of Phase 1/2/3:
- 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.
Exclusion
Key Trial Info
Start Date :
December 30 2021
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
June 30 2028
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT05203679
Start Date
December 30 2021
End Date
June 30 2028
Last Update
July 1 2025
Active Locations (9)
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1
Anhui Provincial Hospital
Hefei, Anhui, China, 230022
2
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China, 100005
3
Nanfang Hospital Southern Medical University
Guangzhou, Guangdong, China, 510515
4
The Second People's Hospital of Shenzhen
Shenzhen, Guangdong, China, 518025