At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 22 enrolled
Drug / intervention
Migalastat HCl 150 mgdrug
Likely dose
Migalastat HCl 150 mgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 12 to <18 Years) With Fabry Disease and Amenable GLA Variants
In Brief
A Phase 3 clinical trial evaluating Migalastat HCl 150 mg for Fabry Disease. Completed, enrolled 22 participants across 8 sites in 2 countries.
Detailed Summary
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.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsFabry Disease
CountriesUnited Kingdom, United States
Collaborators--
Timeline
Phase 3CompletedFinished
20192020202120222023202420252026
First PostedApr 2018
Enrollment StartSep 2018
Primary CompletionFeb 2021
Study CompletionFeb 2021
TodayJul 2026
First PostedApr 17, 2018
Enrollment StartSep 27, 2018
Primary CompletionFeb 2, 2021
Study CompletionFeb 6, 2021
TodayJul 2, 2026
Enrollment to primary: 2.3 yearsPosted 8.2 years ago
Interventions
Migalastat HCl 150 mgdrug
migalastat HCl 150 mg capsule