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Baby-COMET: Avalglucosidase Alfa in Infantile-Onset Pompe Disease
NCT04910776Phase 3Active, not recruiting
- Sponsor
- Sanofi
- Start
- Sep 2021
- Primary completion
- Mar 2026
- Modality
- Replacement Enzymes
- Countries
- 9
01
Overview
Phase 3, open-label, single-arm trial of avalglucosidase alfa in Glycogen storage disease type II (GSD-II), sponsored by Sanofi. Primary endpoint: Proportion of participants who are alive and free of invasive ventilation at 52 weeks. Started Sep 2021; status: active, not recruiting.
02
Drugs
avalglucosidase alfa
03
Conditions
Glycogen storage disease type II (GSD-II)
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