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 study evaluates the safety and tolerability of a single intravenous injection of BBM 002, an adeno-associated virus vector designed to increase levels of factor VIII in patients with Hemophilia A. It targets individuals with low endogenous FVIII activity levels and requires participants to have a history of bleeding events and prior exposure to FVIII products. The study is open-label and involves a single-arm design, focusing on adult males with specific eligibility criteria.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Subjects are fully aware of the purpose, nature, methods and possible adverse reactions of the trial and voluntarily sign informed consent.
2. Males ≥ 18 years of age.
3. Have hemophilia A with ≤2 IU/dL (≤2 %) endogenous FVIII activity levels.
4. Have had ≥150 prior exposure days (EDs) to any recombinant and/or plasma-derived FVIII protein products.
5. Have had bleeding events and/or infusions with FVIII protein products (including recombination and plasma source) during the last 12 weeks documented in the subjects' medical records.
6. Have no prior history of hypersensitivity or anaphylaxis associated with any FVIII or IV immunoglobulin administration.
7. Have no FVIII inhibitor. (eg \<0.6BU/ml Bethesda Units; or the patient's FVIII inhibitor titer was detected \<0.6BU/ml in 2 consecutive times within 1-4 weeks using Bethesda method or Nijmegen method), or no prior medical history of FVIII inhibitor after 150 EDs of FVIII products; no clinical signs or symptoms of decreased response to FVIII products infusion.
8. Agree to use a reliable barrier contraception method from the beginning of signing the informed consent to 52 weeks after BBM002 infusion.
9. Compliance is good, patients and their families have the will of 'gene therapy' clinical trials.

Exclusion Criteria:

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).
2. Currently on antiviral therapy for hepatitis B or C.
3. Patients with coagulation disorders in addition to hemophilia A.
4. Use of any other systematic immunosuppressant other than glucocorticoids within 30 days prior to enrollment.
5. Patients with vaccination history within 30 days prior to screening.
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 by researchers.
7. Patients with known planned major surgery schedule during the 52-week study period aren't eligible.
8. Have participated in a previous gene therapy research trial before screening, or in a clinical study with an investigational drug within 5 half-life of the investigational product, whichever is longer.
9. Have alcohol or drug dependence, or cannot stop drinking throughout the study. 10.Any concurrent clinically significant major disease or condition that the investigator deems unsuitable for participation in the study.

Where it's running

• Institute of Hematology & Blood Diseases Hospital
  Tianjin, Tianjin Municipality, China Recruiting

Study contacts

• Study coordinator: Lei Zhang, MD
• Email: zhanglei1@ihcams.ac.cn
• Phone: 0086-22-23909095

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.

  Recruiting University of Texas Southwestern Medical Center · Dallas, Texas · NCT07692217

• 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.

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• 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.