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Clinical trial

An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants

NCT06904261 is registered in ClinicalTrials.gov with Amicus Therapeutics as lead sponsor.

NCT NCT06904261
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Registry record

As filed in ClinicalTrials.gov.

Title
An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to < 12 Years) With Fabry Disease and Amenable GLA Variants
Lead sponsor
Amicus Therapeutics
Phase
Phase 3
Overall status
Recruiting
Start date
January 8, 2026
Completion date
December 31, 2028
Enrollment
8 (estimated)
Sites
12

Conditions

As the registry lists them; a condition with its own page is linked.

fabry disease