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Clinical Trials in China / NCT06111638
Recruiting Phase 2/3

Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs

NCT06111638 · tracked via the Priya Life Science China tracker
Sponsor
Shanghai Xinzhi BioMed Co., Ltd.
Phase
Phase 2/3
Started
2024-01-03
Last updated
2026-03-17

Condition(s) studied

Hemophilia A

Investigational drug(s) / intervention(s)

Single dose intravenous injection of BBM-H803

Single dose intravenous injection of BBM-H803: Single dose intravenous infusion of BBM-H803, an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene in liver. The dose of BBM-H803 will be calculated according to participant's weight.

Study 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-H803 injection in severe Hemophilia A subjects.

BBM-H803 is an adeno-associated viral (AAV) vector derived from recombinant DNA techniques to contain an expression cassette of the human factor VIII transgene and raises circulating levels of endogenous FVIII.

Eligibility

Sex
MALE
Min age
18 Years
Max age
Healthy volunteers
No
Inclusion Criteria of Phase 1/2/3: 1. Subjects voluntarily sign informed consent form; 2. Males ≥ 18 years; 3. Subjects are clinically diagnosed with severe hemophilia A; 4. Have \> = 150 documented exposure days to a Factor VIII protein product 5. No prior history of hypersensitivity or anaphylaxis associated with any FVIII immunoglobulin; 6. Use a reliable contraception method during the study; 7. Capsid antibody negative; 8. Subjects have good compliance. Exclusion Criteria of Phase 1/2/3: 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, HIV positive patients or Syphilis seropositive patients; 2. Currently on antiviral therapy for hepatitis B or C; 3. Suffer from coagulation disorders other than hemophilia A; 4. In addition to glucocorticoids, any other immunosuppressants are being used before selection; 5. Have vaccination history within 2 months before administration, or vaccination schedule during immunomodulatory therapy; 6. 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; 7. Scheduling of elective major surgery known or planned during the insertion period or the 52-week study period following BBM-H803 infusion; 8. Have participated in a previous gene therapy research trial before screening or have used other test drugs within 4 weeks before screening, or within 5 half-life of the test drug, whichever is longer; Have received emesezumab within 6 months before screening; Or drugs evaluated by the researcher to affect the study; 9. Any herbal preparations (herbal supplements or traditional Chinese medicines of plant, mineral or animal origin) used during the 4 weeks prior to or during the study that may affect liver function, except topical use; Or any Chinese herbal medicine that the researcher evaluates to affect the study; 10. Have alcohol or drug addiction, or cannot stop drinking as advised by the researcher throughout the study; 11. Have acute/chronic infections or other chronic diseases that may pose a risk for the study, or have a major illness, who have a current or previous history of malignant tumors, or who have other unstable medical conditions, including poor mental state and risk of suicide, that the investigator deems unsuitable for participation in the study; 12. Any other conditions that the investigator deems unsuitable for participation in the study.

Primary outcome measure(s)

Trial sites (10)

FacilityCityRegionStatus
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences Beijing Beijing Municipality Recruiting
Southern Hospital, Southern Medical University Guangzhou Guangdong Recruiting
Affiliated Hospital of Guizhou Medical University Guiyang Guizhou Recruiting
Wuhan Union Hospital Affiliated to Huazhong University of Science and Technology Wuhan Hubei Recruiting
Xiangya Hospital Central South University Changsha Hunan Recruiting
The First Affiliated Hospital of Soochow University Suzhou Jiangsu Recruiting
The Affiliated Hospital of Northwest University Xi'an No.3 Hospital Xi’an Shanxi Recruiting
Sichuan Provincial People's Hospital Chengde Sichuan Recruiting
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College Tianjin Tianjin Municipality Recruiting
The Second Affiliated Hospital of Kunming Medical University Kunming Yunnan Recruiting

More Shanghai Xinzhi BioMed Co., Ltd. trials in China

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06111638 on ClinicalTrials.gov ↗ ← All trials in China