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Long-term open-label study of cipaglucosidase alfa plus miglustat in low-body-weight Japanese adults with late-onset Pompe disease
jRCTs061260061Active, not recruiting
- Sponsor
- Amicus Therapeutics
- Start
- Sep 2026
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
- Countries
- 1
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Overview
Open-label, single-arm trial of cipaglucosidase alfa and miglustat in Pompe disease, sponsored by Amicus Therapeutics. Primary endpoints: Safety, Adverse events and 2 more. Started Sep 2026; status: active, not recruiting.
02
Drugs
cipaglucosidase alfamiglustat
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Conditions
Pompe disease
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