Major 5-Year study launches to track Real-World impact of fabry disease treatment
NCT ID NCT06906367
Summary
This study aims to understand the long-term effects of the drug migalastat for adults with Fabry disease, a rare genetic disorder. Researchers will follow 450 patients for up to 5 years to see how well the treatment controls the disease, its safety, and its impact on quality of life compared to other treatments or no treatment. The goal is to gather real-world evidence on the best ways to manage this condition over time.
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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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
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Emory Genetics
RECRUITINGAtlanta, Georgia, 30322, United States
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Lysosomal and Rare Disorders Research and Treatment Center, Inc.
RECRUITINGFairfax, Virginia, 22030, United States
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Renal Disease Research Institute
RECRUITINGDallas, Texas, 75204, United States
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UAB Nephrology Research Clinic at Paula Building
NOT_YET_RECRUITINGBirmingham, Alabama, 35233, United States
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UPMC Children's Hospital of Pittsburgh
NOT_YET_RECRUITINGPittsburgh, Pennsylvania, 15224, United States
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Washington University School of Medicine
NOT_YET_RECRUITINGSt Louis, Missouri, 63110, United States
Conditions
Explore the condition pages connected to this study.