Study reveals risks of stopping thyroid drug in rare genetic disorder
NCT ID NCT05579327
First seen Nov 12, 2025 · Last updated Apr 29, 2026 · Updated 23 times
Summary
This study looked at what happens when males with MCT8 deficiency (a rare genetic condition affecting thyroid hormone transport) stop taking tiratricol. Twenty participants who were stable on the drug were randomly assigned to either continue tiratricol or receive a placebo for 30 days. The goal was to see if stopping treatment caused thyroid hormone levels to rise above normal, requiring rescue therapy.
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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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Addenbrooke's Hospital
Cambridge, United Kingdom
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Erasmus MC
Rotterdam, 3015 GD, Netherlands
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Rare Disease Research, LLC
Kissimmee, Florida, 34746, United States
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Rare Disease Research, LLC
Atlanta, Georgia, 30329, United States
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Rare Disease Research, LLC
Hillsborough, North Carolina, 27278, United States
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SSM Health Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104, United States
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Tranquil Clinical and Research Consulting Services
Webster, Texas, 77598, United States
Conditions
Explore the condition pages connected to this study.