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Open-label study of cipaglucosidase alfa + miglustat in pediatric late-onset Pompe disease
2022-502547-36-00Phase 3Completed
- Sponsor
- Amicus Therapeutics
- Start
- Oct 2023
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
- Countries
- 7
01
Overview
Phase 3, open-label trial of cipaglucosidase alfa and miglustat in Pompe disease (2 cohorts), sponsored by Amicus Therapeutics. Primary endpoints: Incidence of treatment-emergent adverse events, Change in anti-rhGAA antibody titers and 3 more. Started Oct 2023; status: completed.
02
Drugs
cipaglucosidase alfamiglustat
03
Conditions
Pompe disease
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