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.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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22 people
The number who actually took part.
- Started
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Dec 2020
- Expected to finish
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Aug 2027
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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Up to 30 months
- Sex
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Male participants only
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Signed and dated informed consent form from the parents or legal guardian. * Parents stated willingness to comply with all study procedures and availability for the duration of the study. * The participant should be aged between 0 and 30 months on the day of inclusion. * The participant should be male and have a pathogenic mutation in the MCT8 gene. Exclusion Criteria: * Previous treatment with tiratricol. * Previous treatment with LT4 and/or PTU and/or other anti-thyroid medication for a period longer than three months. Patients previously treated with LT4 for a shorter period than 3 months may be included in the study (baseline visit) six weeks (or longer) after last dose of LT4 if two consecutive analyses show stable TFT\*. Patients treated with PTU and/or other anti-thyroid medication for a shorter period than three months may be included in the study (baseline visit) six weeks (or longer) after last dose. * Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency. * Known allergic reactions to components of the IMP. Patients with galactose intolerance, Lapp lactase deficiency or malabsorption of glucose or galactose (the IMP contains lactose). * Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.
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Genom att skicka in godkänner du våra Användarvillkor
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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