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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT05203679
Other study ID # BBM001-CLN1001
Secondary ID
Status Active, not recruiting
Phase Phase 3
First received
Last updated
Start date December 30, 2021
Est. completion date June 30, 2028

Study information

Verified date August 2023
Source Shanghai Belief-Delivery BioMed Co., Ltd
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 32
Est. completion date June 30, 2028
Est. primary completion date June 30, 2024
Accepts healthy volunteers No
Gender Male
Age group 18 Years and older
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

Genetic:
Single dose intravenous injection of BBM-H901
Single dose intravenous infusion of BBM-H901, an adeno-associated virus (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor IX (hFIX) transgene in liver. The dose of BBM-H901 will be 1×10^13 vg/ kg

Locations

Country Name City State
China Peking Union Medical College Hospital Beijing Beijing
China Nanfang Hospital Southern Medical University Guangzhou Guangdong
China Anhui Provincial Hospital Hefei Anhui
China The second Affiliated Hospital of Kunming Medical University Kunming Yunnan
China The Second People's Hospital of Shenzhen Shenzhen Guangdong
China The Second Hospital of Shanxi Medical University Taiyuan Shanxi
China North China University of Science and Technology Affiliated Hospital Tangshan Hebei
China Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College Tianjin Tianjin
China Henan Cancer Hospital Zhengzhou Henan

Sponsors (1)

Lead Sponsor Collaborator
Shanghai Belief-Delivery BioMed Co., Ltd

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Primary Incidence of dose limiting toxicity (DLT) events. To access the numbers of DLT events determined by the Safety Data Review Committee (SRC) within 6 or 10 weeks after administration. 6 or 10 weeks
Primary The incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) To assess the safety of BBM-H901 Injection by TEAEs and SAEs. 52 weeks
Primary Changes in liver function To assess changes in liver function before and after treatment, including alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin (TBIL), alkaline phosphatase (ALP), and gamma-glutamyl transpeptidase (GGT). 52 weeks
Secondary FIX activity All samples collected from participants for plasma FIX activity levels were analyzed and used to determine peak and steady-state vector-derived circulating FIX activity levels. 52 weeks
Secondary FIX antigen level To assess FIX antigen level within 52 weeks after administration. 52 weeks
Secondary The viral load of AAV vector To assess the viral load of AAV vector within 52 weeks after administration. 52 weeks
Secondary Annualized bleeding rate (ABR) To assess ABR, including spontaneous bleeding and traumatic bleeding after administration. 52 weeks
Secondary Improvement in patient's quality of life To assess the proportion of subjects with life improvement from baseline through Hemophilia Joint Health Score (HJHS), Hemophilic Early Arthropathy Detection with UltraSound in China (HEAD-US-C) and the Medical Outcomes Study (MOS) item short from health survey (SF-36) at 4, 12, 26 and 52 weeks. 52 weeks
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