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Cipaglucosidase Alfa/Miglustat in Pediatric Subjects With Late-onset Pompe Disease
NCT03911505Phase 3Active, not recruiting
- Sponsor
- Amicus Therapeutics
- Start
- Feb 2020
- Primary completion
- Mar 2027
- Modality
- Replacement Enzymes
- Countries
- 6
01
Overview
Phase 3, open-label, single-arm trial of cipaglucosidase alfa and miglustat in Pompe disease, sponsored by Amicus Therapeutics. Primary endpoint: Incidence of treatment-emergent adverse events at 52 weeks. Started Feb 2020; status: active, not recruiting.
02
Drugs
cipaglucosidase alfamiglustat
03
Conditions
Pompe disease
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