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Trial intelligence / Profile preview
GC301 gene therapy trial in pediatric late-onset Pompe disease
ChiCTR2200065663Phase 2Not yet recruiting
- Sponsor
- First Medical Center of Chinese PLA General Hospital
- Start
- Dec 2022
- Modality
- Gene Therapies
- Countries
- 1
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Overview
Phase 2, open-label trial of gc301 in Pompe disease (2 arms), sponsored by First Medical Center of Chinese PLA General Hospital. Primary endpoints: Change from baseline in 6-minute walk test, Change from baseline in gross motor function measure-88 total percentage and 1 more. Planned start Dec 2022; status: not yet recruiting.
02
Drugs
gc301
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Conditions
Pompe disease
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