Actively Recruiting
Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
Led by Shanghai Xinzhi BioMed Co., Ltd. · Updated on 2026-03-17
55
Participants Needed
10
Research Sites
386 weeks
Total Duration
On this page
Sponsors
S
Shanghai Xinzhi BioMed Co., Ltd.
Lead Sponsor
S
Shanghai Mianyi Biopharmaceutical Co., Ltd.
Collaborating Sponsor
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male participants aged 18 years or older
- Clinically diagnosed with severe hemophilia A
- Have 150 or more documented exposure days to a Factor VIII protein product
- No prior hypersensitivity or anaphylaxis to any FVIII immunoglobulin
- Use a reliable contraception method during the study
- Negative for capsid antibodies
- Demonstrate good compliance with study requirements
You will not qualify if you...
- Positive for hepatitis B surface antigen or hepatitis B virus DNA
- Positive for hepatitis C virus antibody or RNA
- History of hepatitis B or C only if two negative tests at least 3 months apart are not available
- Positive for HIV or syphilis antibodies
- Currently receiving antiviral therapy for hepatitis B or C
- Have coagulation disorders other than hemophilia A
- Using immunosuppressants other than glucocorticoids before selection
- Vaccinated within 2 months before administration or scheduled for vaccination during immunomodulatory therapy
- Potential liver diseases such as portal hypertension, splenomegaly, hepatic encephalopathy, liver fibrosis stage 3 or higher, liver nodules or cysts, or elevated alpha-fetoprotein deemed clinically significant
- Scheduled for elective major surgery during the study period
- Participation in previous gene therapy trials or use of other test drugs within 4 weeks before screening or within 5 half-lives of the drug
- Use of emesezumab within 6 months before screening
- Use of herbal preparations affecting liver function within 4 weeks before or during the study, except topical use
- Alcohol or drug addiction or inability to stop drinking as advised during the study
- Acute or chronic infections, major illness, history of malignant tumors, unstable medical or mental conditions deemed unsuitable by the investigator
- Any other conditions considered unsuitable for participation by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 10 locations
1
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, China, 100005
Actively Recruiting
2
Southern Hospital, Southern Medical University
Guangzhou, Guangdong, China, 510515
Actively Recruiting
3
Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, China, 550001
Actively Recruiting
4
Wuhan Union Hospital Affiliated to Huazhong University of Science and Technology
Wuhan, Hubei, China, 430000
Actively Recruiting
5
Xiangya Hospital Central South University
Changsha, Hunan, China, 410008
Actively Recruiting
6
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China, 215006
Actively Recruiting
7
The Affiliated Hospital of Northwest University Xi'an No.3 Hospital
Xi’an, Shanxi, China, 710000
Actively Recruiting
8
Sichuan Provincial People's Hospital
Chengde, Sichuan, China, 610031
Actively Recruiting
9
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, China, 300020
Actively Recruiting
10
The Second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, China, 650032
Actively Recruiting
Research Team
W
Wenqi Shao, Master
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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