RecruitingPhase 3NCT06904261

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

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

Sponsor

Amicus Therapeutics

Enrollment

8 participants

Start Date

Jan 8, 2026

Study Type

INTERVENTIONAL

Conditions

Fabry Disease

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

Min Age: 2 YearsMax Age: 11 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a drug called Migalastat HCl 20 mg for people with fabry disease. The study is currently recruiting participants at 11 locations. People eligible for this study include aged 2 Years to 11 Years.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

DRUGMigalastat HCl 20 mg

Migalastat will be supplied as 20-mg dispersible tablets. Migalastat 20-mg dispersible tablets contain 16 mg migalastat free base.

Locations(11)

Emory Genetics

Atlanta, Georgia, United States

University of Minnesota Masonic Children's Hospital

Minneapolis, Minnesota, United States

Atrium Health Levine Children's Hospital

Charlotte, North Carolina, United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Lysosomal and Rare Disorders Research and Treatment Center, Inc.

Fairfax, Virginia, United States

Universitair Ziekenhuis (UZ) Leuven

Leuven, Vlaams-Brabant, Belgium

Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie

Münster, North Rhine-Westphalia, Germany

Hospital Universitario de la Paz

Madrid, Madrid, Spain

Great Ormond Street Hospital for Children NHS Foundation Trust

London, United Kingdom

Manchester University NHS Foundation Trust

Manchester, United Kingdom

View Full Details on ClinicalTrials.gov

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NCT06904261

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