Status:
RECRUITING
Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
Lead Sponsor:
Shanghai Xinzhi BioMed Co., Ltd.
Collaborating Sponsors:
Shanghai MYGT Biopharmaceutical LLC
Conditions:
Hemophilia A
Eligibility:
MALE
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
This is a multi-center, 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 ...
Eligibility Criteria
Inclusion
- Subjects voluntarily sign informed consent form;
- Males ≥ 18 years;
- Subjects are clinically diagnosed with severe hemophilia A;
- Have \> = 150 documented exposure days to a Factor VIII protein product
- No prior history of hypersensitivity or anaphylaxis associated with any FVIII immunoglobulin;
- Use a reliable contraception method during the study;
- Capsid antibody negative;
- Subjects have good compliance.
Exclusion
- 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;
- Currently on antiviral therapy for hepatitis B or C;
- Suffer from coagulation disorders other than hemophilia A;
- In addition to glucocorticoids, any other immunosuppressants are being used before selection;
- Have vaccination history within 2 months before administration, or vaccination schedule during immunomodulatory therapy;
- 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;
- Scheduling of elective major surgery known or planned during the insertion period or the 52-week study period following BBM-H803 infusion;
- 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;
- 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;
- Have alcohol or drug addiction, or cannot stop drinking as advised by the researcher throughout the study;
- 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;
- Any other conditions that the investigator deems unsuitable for participation in the study.
Key Trial Info
Start Date :
January 3 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 30 2030
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT06111638
Start Date
January 3 2024
End Date
June 30 2030
Last Update
July 17 2025
Active Locations (8)
Enter a location and click search to find clinical trials sorted by distance.
1
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, China, 100005
2
Southern Hospital, Southern Medical University
Guangzhou, Guangdong, China, 510515
3
Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, China, 550001
4
Wuhan Union Hospital Affiliated to Huazhong University of Science and Technology
Wuhan, Hubei, China, 430000