NCT06111638 Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
| NCT ID | NCT06111638 |
| Status | Recruiting |
| Phase | Phase 2, Phase 3 |
| Sponsor | Shanghai Xinzhi BioMed Co., Ltd. |
| Condition | Hemophilia A |
| Study Type | INTERVENTIONAL |
| Enrollment | 55 participants |
| Start Date | 2024-01-03 |
| Primary Completion | 2027-05-30 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 55 participants in total. It began in 2024-01-03 with a primary completion date of 2027-05-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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-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 Criteria
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.
Contact & Investigator
Lei Zhang, MD
STUDY CHAIR
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Frequently Asked Questions
Who can join the NCT06111638 clinical trial?
This trial is open to male participants only, aged 18 Years or older, studying Hemophilia A. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06111638 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT06111638 currently recruiting?
Yes, NCT06111638 is actively recruiting participants. Contact the research team at ra@beliefbiomed.com for enrollment information.
Where is the NCT06111638 trial being conducted?
This trial is being conducted at Beijing, China, Guangzhou, China, Guiyang, China, Wuhan, China and 6 additional locations.
Who is sponsoring the NCT06111638 clinical trial?
NCT06111638 is sponsored by Shanghai Xinzhi BioMed Co., Ltd.. The principal investigator is Lei Zhang, MD at Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College. The trial plans to enroll 55 participants.