Plain-English translation of NCT04728841 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Hemophilia A research guide →This study doesn't follow the usual testing phases — it may be an observational study or a different type of research.
This trial is testing a new gene therapy called GS001 for people with severe hemophilia A. The treatment involves a single intravenous infusion that delivers a working copy of the gene your body needs to make clotting factor VIII. Researchers will carefully monitor your safety and measure how well this medication helps your body produce the clotting factor on its own.
Currently, people with severe hemophilia A must receive regular infusions of clotting factor to prevent bleeding. This trial exists to see if a one-time gene therapy can help your body make its own clotting factor, potentially offering a more convenient and lasting solution.
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You would come to the hospital for a single intravenous infusion of the treatment. After that, you would have regular follow-up visits and blood tests over the next 52 weeks to monitor your safety, measure your clotting factor levels, and track how well the treatment is working. The study team will carefully watch for any side effects and adjust future doses based on results from earlier participants.
AI-generated summary from trial data · Jun 6, 2026 · Not medical advice
China
Sponsor
Institute of Hematology & Blood Diseases Hospital, China
Enrollment target
~12 participants
Started
March 2021
Primary completion
July 2028
Age range
18 Years and older
Sex
Male only
Last updated on clinicaltrials.gov in August 2025.
Reach out to the team running this trial. Response times vary — some teams are faster than others.
Central contact
Wei Liu, MD
Chinese Academy of Medical Science and Blood Disease Hospital
Tell us you're interested and we'll help connect you with the research team. We'll walk you through what to expect first — no email needed to get started.