A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Recruiting · NCT06904261 · Registry updated 2026-09-01Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants | Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Cerebral Small Vessel Diseases, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Fabry disease, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, X-linked disease, brain disorder, cardiovascular disorder, central nervous system disorder, cerebrovascular disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis, vascular disorder | Recruiting | Phase 3 | 2026-09-01 | NCT06904261 |
1 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Amicus Therapeutics | 7 |
12 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Atrium Health Levine Children's Hospital | Charlotte | United States | 8 |
| Cincinnati Children's Hospital Medical Center | Cincinnati | United States | 405 |
| Emory Genetics | Atlanta | United States | 2 |
| Great Ormond Street Hospital For Children Nhs Foundation Trust | London | United Kingdom | 23 |
| Hospital Universitario de la Paz | Madrid | Spain | 4 |
| Kitasato University Hospital | Sagamihara | Japan | 12 |
| Lysosomal and Rare Disorders Research and Treatment Center, Inc. | Fairfax | United States | 6 |
| Manchester University NHS Foundation Trust | Manchester | United Kingdom | 22 |
| Universitair Ziekenhuis (UZ) Leuven | Leuven | Belgium | 1 |
| University of Minnesota Masonic Children's Hospital | Minneapolis | United States | 3 |
| Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie | Münster | Germany | 1 |
| UPMC Children's Hospital of Pittsburgh | Pittsburgh | United States | 70 |