A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Start Date
1/8/2026
Completion Date
12/1/2028
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.
Detailed Description
This is a Phase 3b, 2-stage, open-label, uncontrolled, multicenter study to evaluate the safety, PK, PD, and efficacy of 12 months of migalastat treatment in pediatric subjects 2 to \< 12 years of age with Fabry disease and with amenable GLA variants. Subjects must be either naïve to enzyme replacement therapy (ERT) or have stopped ERT at least 14 days before Baseline visit. The study will consist of 2 treatment stages followed by an open-label extension (OLE). Stage 1 will be a treatment period of approximately 3 months (12 weeks); Stage 2 will be a treatment period of 9 months. There will be no break in treatment between Stages 1 and 2. There will be a 30-day (untreated) safety follow-up period for subjects who discontinue treatment at any time. Subjects will be randomly assigned 1:1:1 to 1 of 3 PK sampling groups using interactive response technology (IRT). Four blood samples for the determination of migalastat concentrations in plasma will be collected in one 24-hour period between Day 15 and Day 30 and at Month 6, and 1 PK (trough) sample will be collected at Month 6 and again at Month 12.
Eligibility Criteria
Age Range: 2 years to 11 years
Interventions
Migalastat HCl 20 mg
Conditions
Locations
Emory Genetics
Atlanta, Georgia 30322
United States
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota 55455
United States
Atrium Health Levine Children's Hospital
Charlotte, North Carolina 28203
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio 45229
United States
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania 15224
United States
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia 22030
United States
Universitair Ziekenhuis (UZ) Leuven
Leuven, Vlaams-Brabant 3000
Belgium
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie
Münster, North Rhine-Westphalia 48149
Germany
Hospital Universitario de la Paz
Madrid, Madrid 28046
Spain
Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH
United Kingdom
Manchester University NHS Foundation Trust
Manchester, M13 9WL
United Kingdom