New hope for kids with fabry disease: 12-month drug trial begins
NCT ID NCT06904261
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 29 times
Summary
This study tests a drug called migalastat in 8 children aged 2 to 12 with Fabry disease, a rare genetic condition. The goal is to see if the drug is safe and how it works in the body over 12 months. Participants must have a specific genetic change that makes the drug effective.
Disclaimer
Read more
Show less
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for FABRY DISEASE are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Study contacts
-
Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
-
Atrium Health Levine Children's Hospital
RECRUITINGCharlotte, North Carolina, 28203, United States
-
Cincinnati Children's Hospital Medical Center
RECRUITINGCincinnati, Ohio, 45229, United States
-
Emory Genetics
RECRUITINGAtlanta, Georgia, 30322, United States
-
Great Ormond Street Hospital for Children NHS Foundation Trust
NOT_YET_RECRUITINGLondon, WC1N 3JH, United Kingdom
-
Hospital Universitario de la Paz
RECRUITINGMadrid, Madrid, 28046, Spain
-
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
RECRUITINGFairfax, Virginia, 22030, United States
-
Manchester University NHS Foundation Trust
RECRUITINGManchester, M13 9WL, United Kingdom
-
UPMC Children's Hospital of Pittsburgh
NOT_YET_RECRUITINGPittsburgh, Pennsylvania, 15224, United States
-
Universitair Ziekenhuis (UZ) Leuven
NOT_YET_RECRUITINGLeuven, Vlaams-Brabant, 3000, Belgium
-
University of Minnesota Masonic Children's Hospital
RECRUITINGMinneapolis, Minnesota, 55455, United States
-
Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie
RECRUITINGMünster, North Rhine-Westphalia, 48149, Germany
Conditions
Explore the condition pages connected to this study.