Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
NCT ID: NCT06111638
Last Updated: 2025-07-17
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1/PHASE2
12 participants
INTERVENTIONAL
2024-01-03
2030-06-30
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
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.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Evaluation of the Safety and Efficacy of Hemophilia B Gene Therapy Drug
NCT05203679
Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
NCT05709288
Gene Therapy for Chinese Hemophilia B
NCT04135300
Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
NCT06238908
To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
NCT06833983
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Arm of BBM-H803
The dose of BBM-H803 will be calculated according to participant's weight
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.
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
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.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
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
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.
18 Years
MALE
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Shanghai MYGT Biopharmaceutical LLC
UNKNOWN
Shanghai Xinzhi BioMed Co., Ltd.
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Lei Zhang, MD
Role: STUDY_CHAIR
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
Beijing, Beijing Municipality, China
Southern Hospital, Southern Medical University
Guangzhou, Guangdong, China
Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou, China
Wuhan Union Hospital Affiliated to Huazhong University of Science and Technology
Wuhan, Hubei, China
Xiangya Hospital Central South University
Changsha, Hunan, China
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, China
The Second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, China
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
BBM002-CLN1001
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.