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GC301 AAV gene therapy in infants and young children with Pompe disease
ChiCTR2200065664Phase 2Completed
- Sponsor
- First Medical Center of Chinese PLA General Hospital
- Start
- Oct 2022
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of gc301 in Pompe disease, sponsored by First Medical Center of Chinese PLA General Hospital. Primary endpoints: Change in GMFM-88 total percentage at week 52 vs baseline and Change in left ventricular mass (LVM) Z-score at week 52 vs baseline. Started Oct 2022; status: completed.
02
Drugs
gc301
03
Conditions
Pompe disease
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