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Expanded access study of cipaglucosidase alfa + miglustat for late-onset Pompe disease in Japanese adults
jRCT2061210056Active, not recruiting
- Sponsor
- Amicus Therapeutics
- Start
- Dec 2021
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Open-label, single-arm trial of cipaglucosidase alfa in Pompe disease, sponsored by Amicus Therapeutics. Primary endpoint: Safety of ATB200/AT2221 co-administration. Started Dec 2021; status: active, not recruiting.
02
Drugs
cipaglucosidase alfa
03
Conditions
Pompe disease
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