Gene therapy for treating Hemophilia A

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This clinical trial evaluates the safety and efficacy of a single intravenous dose of BBM-H803, a gene therapy designed to increase levels of factor VIII in patients with severe Hemophilia A. The study is multi-center and open-label, focusing on heavy Hemophilia A subjects who meet specific inclusion criteria. Participants will be monitored for safety, tolerability, and the effectiveness of the treatment in raising endogenous FVIII levels.

Why it matters

Who can join: the full rules

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.

Where it's running

• Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
  Beijing, Beijing Municipality, China Recruiting
• Southern Hospital, Southern Medical University
  Guangzhou, Guangdong, China Recruiting
• Affiliated Hospital of Guizhou Medical University
  Guiyang, Guizhou, China Recruiting
• Wuhan Union Hospital Affiliated to Huazhong University of Science and Technology
  Wuhan, Hubei, China Recruiting
• Xiangya Hospital Central South University
  Changsha, Hunan, China Recruiting
• The First Affiliated Hospital of Soochow University
  Suzhou, Jiangsu, China Recruiting

Study contacts

• Study coordinator: Wenqi Shao, Master
• Email: ra@beliefbiomed.com
• Phone: 13636317441

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• First‑in‑human test of ARD001 in healthy adults and people with hemophilia A

  This study tests ARD001 to see if it is safe and tolerable in healthy adults and in people with hemophilia A, using a single dose (Phase 1) and potentially repeated doses (Phase 2) if results look good.

  Recruiting Phase 1/2 Arditus Pte Ltd · Singapore · NCT07787091

• Hemophilia A: comparing long-acting FVIII replacement vs non-FVIII therapy in kids

  This study tests whether children with moderate to severe Hemophilia A on long-acting FVIII or non-FVIII therapies form clots differently and develop small joint bleeds or early joint changes.

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• SR604: a new treatment tested in adults with Hemophilia A/B or congenital Factor VII deficiency

  This study tests a single and multiple doses of SR604 to see if it is safe and effective for adults with Hemophilia A or B, with or without inhibitors, and for those with congenital Factor VII deficiency.

  Recruiting at 7 sites Phase 1/2 Shanghai RAAS Blood Products Co., Ltd. · Changsha and 8 other locations · NCT07644832

• Bone health in severe hemophilia A: emicizumab vs efanesoctocog alfa

  This study tests whether bone density and bone health markers differ in adults with severe hemophilia A who use emicizumab versus efanesoctocog alfa for prophylaxis.

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• Joint health, balance, and quality of life in adults with hemophilia A

  See how joint damage and postural balance relate to quality of life in adults with severe hemophilia A.

  Recruiting Yuzuncu Yil University · Van · NCT07523399

• NXT007 versus emicizumab to prevent bleeds in people with Hemophilia A

  This will test whether NXT007 prevents bleeding better than emicizumab in people aged 12 and older with Hemophilia A, including those with or without factor VIII inhibitors.

  Recruiting at 63 sites Phase 3 Hoffmann-La Roche · Orange, California and 62 other locations · NCT07416604

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.