New hope for kids with fabry: migalastat trial opens
NCT ID NCT06904261
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.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Atrium Health Levine Children's Hospital
RECRUITINGCharlotte, North Carolina, 28203, United States
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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
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
RECRUITINGLeuven, Vlaams-Brabant, 3000, Belgium
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University of Minnesota Masonic Children's Hospital
RECRUITINGMinneapolis, Minnesota, 55455, United States
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Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie
RECRUITINGMünster, North Rhine-Westphalia, 48149, Germany