Study design & results
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Trial intelligence / Profile preview
One-year alglucosidase alfa in Chinese patients with late-onset Pompe disease
2024-000461-24Phase 4
- Sponsor
- Sanofi
- Start
- Dec 2024
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
01
Overview
Phase 4, open-label, single-arm trial of alglucosidase alfa in Pompe disease, sponsored by Sanofi. Primary endpoints: Change from baseline in Six-minute walk test and Change from baseline in predicted forced vital capacity.
02
Drugs
alglucosidase alfa
03
Conditions
Pompe disease
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