Status:
RECRUITING
Gene Therapy for Chinese Hemophilia A
Lead Sponsor:
Institute of Hematology & Blood Diseases Hospital, China
Conditions:
Hemophilia A
Gene Therapy
Eligibility:
MALE
18+ years
Phase:
NA
Brief Summary
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 FV...
Detailed Description
IHBDH-GTHA-2020 is a open- label, non- randomized study to evaluate the safety, tolerability and kinetics of GS001 in hemophilia A subjects with residual FVIII levels\<1 IU/dl. The first patient will ...
Eligibility Criteria
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
- 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;
- Currently Receiving antiviral therapy for hepatitis B and C;
- Have history of chronic infections or other chronic diseases that may pose a risk to the study participation;
- 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;
- 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;
- Major surgery planned in 1 year period following the infusion with GS001;
- 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;
- Patients who are unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol;
- Evidence of other bleeding disorders not associated with hemophilia A.
Key Trial Info
Start Date :
March 4 2021
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
July 31 2028
Estimated Enrollment :
12 Patients enrolled
Trial Details
Trial ID
NCT04728841
Start Date
March 4 2021
End Date
July 31 2028
Last Update
August 1 2025
Active Locations (1)
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1
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, Tianjin Municipality, China, 300020