New hope for kids with rare genetic disorder
NCT ID NCT06904261
Summary
This study is testing a medication called migalastat in children aged 2 to under 12 years old who have Fabry disease, a rare genetic condition. The goal is to see if the drug is safe, how the body processes it, and if it helps control the disease over 12 months of treatment. The study is open to children who are either new to treatment or have stopped a previous therapy.
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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
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Emory Genetics
RECRUITINGAtlanta, Georgia, 30322, United States
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Great Ormond Street Hospital for Children NHS Foundation Trust
NOT_YET_RECRUITINGLondon, WC1N 3JH, United Kingdom
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Hospital Universitario de la Paz
NOT_YET_RECRUITINGMadrid, Madrid, 28046, Spain
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Lysosomal and Rare Disorders Research and Treatment Center, Inc.
RECRUITINGFairfax, Virginia, 22030, United States
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Manchester University NHS Foundation Trust
RECRUITINGManchester, M13 9WL, United Kingdom
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UPMC Children's Hospital of Pittsburgh
NOT_YET_RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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Universitair Ziekenhuis (UZ) Leuven
NOT_YET_RECRUITINGLeuven, Vlaams-Brabant, 3000, Belgium
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Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie
NOT_YET_RECRUITINGMünster, North Rhine-Westphalia, 48149, Germany
Conditions
Explore the condition pages connected to this study.