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ATB200 + AT2221 long-term extension in adults with late-onset Pompe disease
jRCT2011200003Phase 3Completed
- Sponsor
- Amicus Therapeutics
- Start
- Oct 2020
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
01
Overview
Phase 3, open-label, single-arm trial of cipaglucosidase alfa and miglustat in Pompe disease, sponsored by Amicus Therapeutics. Primary endpoint: Proportion of participants with treatment-emergent adverse events. Started Oct 2020; status: completed.
02
Drugs
cipaglucosidase alfamiglustat
03
Conditions
Pompe disease
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