New study tracks Long-Term safety of rare disease treatment
NCT ID NCT04632953
First seen Nov 01, 2025 · Last updated May 12, 2026 · Updated 26 times
Summary
This study follows 150 people with long-chain fatty acid oxidation disorders (LC-FAOD) to monitor the long-term safety of the drug triheptanoin. Researchers will track serious side effects, pregnancy outcomes, and infant health. The goal is to understand how well the treatment works over time and ensure it remains safe for patients.
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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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Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital
Chicago, Illinois, 60611, United States
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Boston Children's Hospital
Boston, Massachusetts, 02215, United States
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CHEO (Children's Hospital Eastern Ontario)
Ottawa, Ontario, K1H 8L1, Canada
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Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
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Columbia University
New York, New York, 10032, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Seattle Children's Hospital
Seattle, Washington, 98020, United States
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SickKids (The Hospital for Sick Children)
Toronto, Ontario, M5G 1X8, Canada
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The Emory Clinic
Atlanta, Georgia, 30322, United States
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University of Alberta
Edmonton, Alberta, T6G 1C9, Canada
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University of California San Francisco
San Francisco, California, 94158, United States
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University of Minnesota
Minneapolis, Minnesota, 55454, United States
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University of South Florida
Tampa, Florida, 33606, United States
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University of Utah
Salt Lake City, Utah, 84108, United States
Conditions
Explore the condition pages connected to this study.