A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Condition(s) studied
Investigational drug(s) / intervention(s)
Migalastat HCl 20 mg: Migalastat will be supplied as 20-mg dispersible tablets. Migalastat 20-mg dispersible tablets contain 16 mg migalastat free base.
Study summary
An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants.
Eligibility
Primary outcome measure(s)
- Safety: Incidence of TEAEs, SAEs, and AEs leading to discontinuation of study drug — Day 1 (after dosing) through Month 12 and follow-up (30 days after last dose)
- Pharmacokinetics (PK): Maximum Observed Plasma Concentration (Cmax) of Migalastat — 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12
- Pharmacokinetics (PK): Minimum Observed Plasma Concentration (Cmin) of Migalastat — 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12
- Pharmacokinetics (PK): Area Under The Plasma Concentration-time Curve Over The Dosing Interval (AUCtau) of Migalastat — 0 to 12 hours postdose during the first month of study and trough samples at Months 6 and 12
Trial sites (12)
| Facility | City | Region | Status |
|---|---|---|---|
| Emory Genetics | Atlanta | Georgia | Recruiting |
| University of Minnesota Masonic Children's Hospital | Minneapolis | Minnesota | Recruiting |
| Atrium Health Levine Children's Hospital | Charlotte | North Carolina | Recruiting |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | Recruiting |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | Pennsylvania | Recruiting |
| Lysosomal and Rare Disorders Research and Treatment Center, Inc. | Fairfax | Virginia | Recruiting |
| Universitair Ziekenhuis (UZ) Leuven | Leuven | Vlaams-Brabant | Recruiting |
| Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie | Münster | North Rhine-Westphalia | Recruiting |
| Kitasato University Hospital | Sagamihara | Kanagawa | Not Yet Recruiting |
| Hospital Universitario de la Paz | Madrid | Madrid | Recruiting |
| Great Ormond Street Hospital for Children NHS Foundation Trust | London | United Kingdom | Not Yet Recruiting |
| Manchester University NHS Foundation Trust | Manchester | United Kingdom | Recruiting |
More Amicus Therapeutics trials in Spain
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06904261 on ClinicalTrials.gov ↗ ← All trials in Spain