Plain-English translation of NCT03217032 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Hemophilia A research guide →Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This trial is testing a lentiviral gene therapy designed to treat hemophilia A, a bleeding disorder where the body cannot produce enough clotting factor VIII. Researchers will take your own stem cells, add a working copy of the clotting factor VIII gene using advanced gene delivery technology, and return them to your body so you can produce clotting factor on your own. The goal is to reduce or eliminate the need for repeated factor VIII infusions.
Current hemophilia A treatment requires lifelong intravenous infusions of clotting factor, which is expensive and inconvenient but still doesn't cure the disease. This trial exists to see whether gene therapy can help your body make its own clotting factor, potentially offering a one-time or long-lasting solution.
You likely qualify if…
You likely don't qualify if…
If accepted, researchers will collect your stem cells and add a working clotting factor VIII gene to them in the laboratory. You will then receive an infusion of these modified cells back into your body. The study will monitor your safety and whether your body starts producing clotting factor VIII on its own, with regular blood tests and visits to track your bleeding episodes and overall health.
AI-generated summary from trial data · Jul 2, 2026 · Not medical advice
China
Phase
Safety & dosing
Sponsor
Shenzhen Geno-Immune Medical Institute
Enrollment target
~10 participants
Started
June 2025
Primary completion
May 2027
Age range
2 Years – 65 Years
Last updated on clinicaltrials.gov in June 2026.
Reach out to the team running this trial. Response times vary — some teams are faster than others.
Central contact
Lung-Ji Chang, PhD
Shenzhen Geno-Immune Medical Institute
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.