Gene Therapy Tested in Adults with Hemophilia A
Tests a single infusion of GS001 gene therapy in adults with hemophilia A.
Plain-language title generated from the registry record
Registry title: Gene Therapy for Chinese Hemophilia A
Key facts
- Where
- Tianjin, ChinaSee the site
Where in China
- Chinese Academy of Medical Science and Blood Disease HospitalTianjin, ChinaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Institute of Hematology & Blood Diseases Hospital, China
- Trial code
- NCT04728841
- Record updated
- 1 August 2025
- Original description
IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with <1 IU/dl residual FVIII levels.Read full original descriptionShow less
Clinical Exploration of Clinical Exploration Adeno-associated Virus Vector Expression of Human Coagulation Factor VIII Gene Therapy for Hemophilia A
IHBDH-GTHA-2020 is an open- label, non- randomized study to evaluate the safety, tolerability and kinetics of a single intravenous infusion of GS001 in hemophilia A subjects with <1 IU/dl residual FVIII levels.
Inclusion: Be able to understand the purpose and risks of the study and provide informed consent according to national and local privacy laws; Male subjects and ≥ 18 years of age; 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 ; No history of hypersensitivity or anaphylaxis associated with FVIII product administration; 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 ; 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; 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: Currently Receiving antiviral therapy for hepatitis B and C; 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; The subject has any concurrent diseases that cannot tolerate treatments of prednisone or prednisolone as judged by the investigator; Major surgery planned in 1 year period following the infusion with GS001; 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; Have history of chronic infections or other chronic diseases that may pose a risk to the study participation; History of arterial or venous thromboembolic events (e.g., deep vein thrombosis, non-hemorrhagic stroke, pulmonary embolism, myocardial infarction, arterial embolism); Known inherited or acquired thrombophilia, including conditions associated with increased risk of thromboembolism, such as atrial fibrillation; Hypersensitivity to the study vector; 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; Evidence of other bleeding disorders not associated with hemophilia A; Patients who are unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol.