Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)
Completed · Phase 3
Conditions studied: Fabry Disease
In brief
This was an open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric participants 12 to \<18 years of age with Fabry disease and amenable gene encoding α-galactosidase A (GLA) variants.
Key facts
- Study ID
- NCT03500094
- Run by
- Amicus Therapeutics
- People needed
- 22
- Starts
- 2018-09-27
- Expected to finish
- 2021-02-06
- Last updated by the study team
- 2021-11-30
Who can join
Age: 12 and older, up to 17. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Clinical Study Site — Tampa, Florida, United States
- Clinical Study Site — Atlanta, Georgia, United States
- Clinical Study Site — Minneapolis, Minnesota, United States
- Clinical Study Site — Columbia, Missouri, United States
- Clinical Study Site — Cincinnati, Ohio, United States
- Clinical Study Site — Pittsburgh, Pennsylvania, United States
- Clinical Study Site — Fairfax, Virginia, United States
- Clinical Study Site — London, United Kingdom
Full record on ClinicalTrials.gov
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