NCT02396459|Active Not RecruitingPhase 2DMCFDA Drug
Triac Trial II in MCT8 Deficiency Patients
Tiratricol Treatment of Children With Monocarboxylate Transporter 8 Deficiency: Triac Trial II
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Sponsor study identifier
interventional
22
4 countries
5
Study Start
December 7, 2020
Actual (reported)Primary Completion
July 18, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
August 18, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
This study will investigate the effect of treatment with tiratricol (also called Triac) in young boys (≤30 months) with MCT8 deficiency (also called the Allan-Herndon-Dudley syndrome (AHDS)). The hypothesis tested is that treatment with tiratricol will have a beneficial effect on the hypothyroid state in the brain as well as the hyperthyroid state in peripheral organs and tissues in these patients. Patients will initially be treated for 96 weeks with tiratricol, treatment effect on neurodevelopment impairment caused by hypothyroidism and peripheral thyrotoxicosis will be evaluated after 96 weeks treatment. Patients will be offered to continue on treatment for an additional 3 years.
Registry-reported information. Elapsed time and enrollment totals alone do not measure study progress or recruitment pace.
August 18, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
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Dates and qualifiers reported by the registry. Estimated dates do not confirm that a milestone has occurred.
March 7, 2015
Actual (reported)March 24, 2015
Actual (reported)December 7, 2020
Actual (reported)July 18, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
August 18, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Gross Motor Function Measure 88 (GMFM 88) total score
To evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, measured by the Gross Motor Function Measure (GMFM)-88 assessment. Potential result values range from 0 to 100%, the latter being representative for a 4-year old healthy child. A high score is equivalent to better/more neurodevelopment and is therefore a better outcome than a low score.
96 weeks, Year 3, Year 4 and Year 5
Bayley Scales of Infant Development III Gross Motor Skill Domain score
To evaluate the effect of tiratricol treatment on neurodevelopment measured by the Bayley Scales of Infant Development (BSID-III) Gross Motor Skill Domain score. Potential total raw scores range from 0-72, and can be age-adjusted before analysis. A high score is equivalent to better/more neurodevelopment and is therefore a better outcome than a low score
96 weeks, Year 3, Year 4 and Year 5
GMFM-88 individual item score 10 and 24.
96 weeks, Year 3, Year 4 and Year 5
Bayley Scales of Infant Development III score.
96 weeks, Year 3, Year 4 and Year 5
Serum T3 concentrations
96 weeks, Year 3, Year 4 and Year 5
Tissue-specific markers of thyroid state: serum sex-hormone binding globulin concentrations for liver
96 weeks, Year 3, Year 4 and Year 5
Tissue-specific markers of thyroid state: serum creatine kinase concentrations for muscles
96 weeks, Year 3, Year 4 and Year 5
Patient Quality of Life by Infant Toddler Quality of Life Questionnaire (ITQoL)
96 weeks and Year 5
Parent Quality of Life by Parenting Stress index (PSI-4 SF)
96 weeks and Year 5
Evaluate the effect of tiratricol treatment on neurological symptoms (Hammersmith Infant Neurological Exam, HINE)
96 weeks, Year 3, Year 4 and Year 5
Tiratricol (Triac) treatment
Tiratricol, individually titrated dose
These entries preserve how the registry describes the intervention. Their treatment roles are shown separately from molecular identities.
Formulated product
Tiratricol
The registry describes a specific formulation. Its exact chemical ingredients and approval status require separate evidence.
TABLET
View registry recordFormulated product
Tiratricol
The registry describes a specific formulation. Its exact chemical ingredients and approval status require separate evidence.
TABLET
View registry recordThe registry names these ingredients. The formulation’s exact chemical form has not been confirmed.
Tiratricol
Named ingredient: Tiratricol
View registry recordTiratricol
Named ingredient: Tiratricol
View registry recordOregon Health & Science University (OHSU) Doernbecher Childrens Hospital
Portland, Oregon, 97239, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Charles University and Motol University Hospital; The department of peadiatrics of the 2nd faculty of medicine
Prague, 15006, Czechia
Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie
Berlin, 13353, Germany
Erasmus MC
Rotterdam, 3015 GD, Netherlands
W.E. Visser, MD, PhD
Erasmus Medical Center
Kristina Sjöblom Nygren, MD
Rare Thyroid Therapeutics International AB
Lindsey Nicol
Oregon Health& Science University (OHSU) Doernbecher Childrens Hospital
Jan Lebl
Charles University and Motol University Hospital
22 participants (actual enrollment reported by the registry).
First Submitted
March 7, 2015
First Posted
March 24, 2015
Study Start
December 7, 2020
Actual (reported)Primary Completion
Register for Patients With Thyroid Hormone Resistance.
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Heiko Krude
Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie
Andrew Bauer, MD
Children's Hospital of Philadelphia
July 18, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Study Completion
August 18, 2027
EstimatedPlanned date reported by the registry; this milestone is not confirmed as achieved.
Last Updated
February 20, 2026
Record last verified: 2026-02
Because of the rarity of MCT8 deficiency and the small number of patients in this trial, individual patient data beyond what is reported in the upcoming manuscript will not be shared to safeguard patient privacy.
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