Fabry disease patients monitored for Real-World treatment effects
NCT ID NCT06906367
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study follows 450 US adults with Fabry disease who are taking migalastat or enzyme replacement therapy. Researchers will track kidney function, heart and brain events, and quality of life over time to see how well these treatments work in everyday practice.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
migalastat HCl and enzyme replacement therapy (ERT)
What this could lead to
If successful, this study could provide real-world evidence on how well migalastat works over time for Fabry disease, potentially guiding better treatment decisions.
What could go wrong
This is an observational study, not a controlled trial, so results may be influenced by patient differences. It does not test a new treatment, only monitors existing ones.
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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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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New York-Presbyterian Morgan Stanley Children's Hospital - Columbia University Medical Center
NOT_YET_RECRUITINGNew York, New York, 10032, 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
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