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Open-label 12-month migalastat treatment in pediatric Fabry disease (amenable GLA variants)
jRCT2031260371Recruiting
- Sponsor
- Amicus Therapeutics
- Start
- Aug 2026
- Modality
- Classical Binding Small Molecules → Small Molecules
01
Overview
Open-label, single-arm trial of migalastat in Fabry disease (FD), sponsored by Amicus Therapeutics. Primary endpoints: Safety: Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events (AEs) leading to discontinuation of study drug, Pharmacokinetics: Maximum observed plasma concentration (Cmax) of migalastat and 2 more. Started Aug 2026; status: recruiting.
02
Drugs
migalastat
03
Conditions
Fabry disease (FD)
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