Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial

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The hallmarks of MCT8 deficiency are severe psychomotor retardation due to intracellular hypothyroidism in neuronal tissues and peripheral T3 thyrotoxicosis associated low weight, muscle wasting, high normal or increased heart rate and systolic blood pressure. TRIAC – the T3 analogue 3,3’,5-tri-iodothyroacetic acid – enters target cells in an MCT8 independent way, and therefore could potentially improve intracellular thyroid hormone deficiency in the brain. On the other hand, TRIAC has only weak thyromimetic activity in peripheral tissues, thus represents a bona fide candidate to improve the MCT8 deficiency associated imbalance between neuronal intracellular hypothyroidism and peripheral hyperthyroidism.