Plain-English translation of NCT06391736 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This trial is testing GC301, a new gene therapy designed to treat late-onset Pompe disease by delivering a healthy gene directly into your body through an injection. The goal is to help your muscles work better and reduce the weakness that comes with this condition. Researchers want to learn if the treatment is safe and whether it actually helps patients feel stronger and move more easily.
Late-onset Pompe disease causes muscles to gradually weaken over time, and current treatments don't work well enough for everyone. This trial exists to see if this new gene therapy approach could offer a better option by addressing the root cause of the disease.
You likely qualify if…
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You will receive a single intravenous injection of the study medication. The trial will compare two different dose levels to see which works best and is safest. After the injection, you'll have regular appointments to check your muscle strength, breathing, and walking ability, and to monitor for any side effects. The study team will follow your progress over time to see if the treatment helps you feel stronger and improves your daily functioning.
AI-generated summary from trial data · Aug 29, 2026 · Not medical advice
China
Phase
Safety & dosing / Early efficacy
Sponsor
GeneCradle Inc
Enrollment target
~33 participants
Started
April 2024
Primary completion
December 2026
Age range
6 Years and older
Last updated on clinicaltrials.gov in July 2025.
Reach out to the team running this trial. Response times vary — some teams are faster than others.
Central contact
GeneCradle, Inc China
Chinese PLA General 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.