New hope for kids with fabry: migalastat trial opens

NCT ID NCT06904261

Recruiting now Disease control Sponsor: Amicus Therapeutics Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests the drug migalastat in 8 children aged 2 to 12 with Fabry disease, a rare genetic disorder. The goal is to see if the drug is safe, how it moves through the body, and if it helps protect kidney function. Participants will take the medicine for 12 months.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Migalastat (Galafold)
What this could lead to: If successful, this could provide a treatment option for children with Fabry disease, potentially slowing disease progression and improving quality of life.
What could go wrong: This is a small, early-phase study with only 8 participants, so results may not apply to all patients. The drug may cause side effects or not work as well in children as in adults.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Fabry disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

FABRY DISEASE

Contacts and locations

Locations

Atrium Health Levine Children's Hospital
RECRUITING

Charlotte, North Carolina, 28203, United States
Cincinnati Children's Hospital Medical Center
RECRUITING

Cincinnati, Ohio, 45229, United States
Emory Genetics
RECRUITING

Atlanta, Georgia, 30322, United States
Great Ormond Street Hospital for Children NHS Foundation Trust
NOT_YET_RECRUITING

London, WC1N 3JH, United Kingdom
Hospital Universitario de la Paz
RECRUITING

Madrid, Madrid, 28046, Spain
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
RECRUITING

Fairfax, Virginia, 22030, United States
Manchester University NHS Foundation Trust
RECRUITING

Manchester, M13 9WL, United Kingdom
UPMC Children's Hospital of Pittsburgh
NOT_YET_RECRUITING

Pittsburgh, Pennsylvania, 15224, United States
Universitair Ziekenhuis (UZ) Leuven
RECRUITING

Leuven, Vlaams-Brabant, 3000, Belgium
University of Minnesota Masonic Children's Hospital
RECRUITING

Minneapolis, Minnesota, 55455, United States
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie
RECRUITING

Münster, North Rhine-Westphalia, 48149, Germany

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