Experimental drug shows promise for rare genetic syndrome in toddlers
NCT ID NCT02396459
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2 trial tests the drug tiratricol (Triac) in 22 young boys (up to 30 months old) with MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome. The goal is to see if the drug can improve brain development and reduce harmful thyroid effects in the body. Participants receive tiratricol for 96 weeks, with an option to continue for 3 more years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- Tiratricol (also called Triac)
- What this could lead to
- If successful, this could point toward a treatment that improves motor skills and brain development in young boys with this rare, severe disorder.
- What could go wrong
- This is a small, early-phase trial with only 22 participants, so results may not apply to all patients. The treatment may not work or could have side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
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
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