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Phase 3 study of cipaglucosidase alfa + miglustat versus alglucosidase alfa + placebo in adults with late-onset Pompe disease
2018-000755-40Phase 3Completed
- Sponsor
- Amicus Therapeutics
- Start
- Nov 2018
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Small Molecules
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial in Pompe disease comparing cipaglucosidase alfa + miglustat with alglucosidase alfa + placebo (2 arms). Primary endpoint: Change from baseline to Week 52 in 6-minute walk distance. Started Nov 2018; status: completed.
02
Drugs
cipaglucosidase alfamiglustat
03
Conditions
Pompe disease
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