New hope for kids with rare genetic disease

NCT ID NCT06904261

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

Summary

This study is testing the safety and effectiveness of a drug called migalastat in children aged 2 to 12 who have Fabry disease, a rare genetic condition. The goal is to see how the children's bodies process the drug and if it helps control their disease over 12 months of treatment. Participants will take the drug regularly and be monitored for side effects and changes in their health.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Vår säkerhetsrekommendation!

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

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
NOT_YET_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
NOT_YET_RECRUITING

Leuven, Vlaams-Brabant, 3000, Belgium
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie
NOT_YET_RECRUITING

Münster, North Rhine-Westphalia, 48149, Germany

Conditions

Explore the condition pages connected to this study.

FABRY DISEASE