Gene Therapy for Chinese Hemophilia A
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexMale
Age18+
SponsorInstitute of Hematology & Blood Diseases Hospital, China
About this trial
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.
Eligibility criteria
Qualifiers
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;
Disqualifiers
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;
Trial design
Treatments tested in this trial
- Injection of GS001
Treatment groups
12 Participants
are divided into 1 treatment groupSponsors and collaborators
Source ClinicalTrials.gov