Promising drug trial for rare genetic disorder in toddlers
NCT ID NCT02396459
First seen Mar 02, 2026 · Last updated Apr 30, 2026 · Updated 6 times
Summary
This study tests a drug called tiratricol (Triac) in 22 young boys (up to 30 months old) with MCT8 deficiency, a rare genetic disorder that causes severe brain and thyroid problems. The goal is to see if the drug can improve brain development and reduce harmful thyroid effects. Boys receive treatment for 96 weeks, with an option to continue for 3 more years.
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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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Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie
Berlin, 13353, Germany
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Charles University and Motol University Hospital; The department of peadiatrics of the 2nd faculty of medicine
Prague, 15006, Czechia
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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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Oregon Health & Science University (OHSU) Doernbecher Childrens Hospital
Portland, Oregon, 97239, United States
Conditions
Explore the condition pages connected to this study.