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Trial intelligence / Profile preview
POTENTIAL long-term follow-up study in Pompe disease
NL-OMON61671Phase 4Not yet recruiting
- Sponsor
- Erasmus MC
- Start
- Sep 2026
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Countries
- 1
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Overview
Phase 4, open-label, single-arm trial of avalglucosidase alfa in Pompe disease, sponsored by Erasmus MC. Primary endpoints: Alberta Infant Motor Scale, Quick Motor Function Test and 3 more. Planned start Sep 2026; status: not yet recruiting.
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Drugs
avalglucosidase alfa
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Conditions
Pompe disease
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