Gene therapy for hemophilia A in adults

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 study evaluates the safety, tolerability, and kinetics of a single intravenous infusion of GS001 in adult patients with hemophilia A who have very low levels of FVIII. It is an open-label, non-randomized trial where the first patient will receive a specific dose of GS001, followed by adjustments based on safety assessments and FVIII activity levels. The study aims to determine the appropriate dosing and monitor the effects of the gene therapy over time.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Be able to understand the purpose and risks of the study and provide informed consent according to national and local privacy laws;
2. Male subjects and ≥ 18 years of age;
3. Have hemophilia A with ≤1 IU/dL (≤1%) endogenous FVIII activity levels at the time of screening. If the screening result is \>1% due to previous treatment with FVIII product, then it may be confirmed by documented historical evidence from a certified clinical laboratory demonstrating ≤1% FVIII activity levels ;
4. No history of hypersensitivity or anaphylaxis associated with FVIII product administration;
5. Have no measurable FVII inhibitor as assessed by laboratory two times that were at least one week apart; or documented no prior history of FVIII inhibitor after 150 EDs and no clinical signs or symptoms of decreased response to FVIII infusion ;
6. Have acceptable laboratory values sampled at screening and repeated prior to Day 0; A. Hemoglobin ≥ 11 g/dL; B. Platelets ≥ 100 x 10\^9/L; C. AST, ALT, alkaline phosphatase ≤ 1.25 upper limit of normal (ULN); D. Bilirubin ≤ 1.25 ULN; E. Creatinine ≤ 2 mg/dL.
7. Agree to use reliable barrier contraception until the end of the 52 weeks observation period, and three consecutive semen samples are negative for vector sequences after GS001 infusion.

Exclusion Criteria:

1. Have Hepatitis B, hepatitis C or HBsAg, HCVAb, HBV-DNA, HCV-RNA are positive and have clinical significance. Both natural clearers and those who have cleared HCV on antiviral therapy are deemed eligible;
2. Currently Receiving antiviral therapy for hepatitis B and C;
3. Have history of chronic infections or other chronic diseases that may pose a risk to the study participation;
4. Have participated in a previous gene therapy research trial within the last 52 weeks or in a clinical study with an investigational drug within the past 30 days;
5. The subject has any concurrent diseases that cannot tolerate treatments of prednisone or prednisolone as judged by the investigator;
6. History of arterial or venous thromboembolic events (e.g., deep vein thrombosis, non-hemorrhagic stroke, pulmonary embolism, myocardial infarction, arterial embolism);
7. Known inherited or acquired thrombophilia, including conditions associated with increased risk of thromboembolism, such as atrial fibrillation;
8. Major surgery planned in 1 year period following the infusion with GS001;
9. Hypersensitivity to the study vector;
10. Have clinically major diseases or any other unspecified conditions that, in the opinion of the Investigator, makes the subject unsuitable for participating in the study;
11. Patients who are unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol;
12. Evidence of other bleeding disorders not associated with hemophilia A.

Where it's running

• Chinese Academy of Medical Science and Blood Disease Hospital
  Tianjin, Tianjin Municipality, China Recruiting

Study contacts

• Principal investigator: Lei Zhang, MD — Chinese Academy of Medical Science and Blood Disease Hospital
• Study coordinator: Wei Liu, MD
• Email: liuwei1@ihcams.ac.cn
• Phone: +82223909240

Similar studies

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

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

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• 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.

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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.