Tiratricol Treatment of Children with Monocarboxylate Transporter 8 Deficiency: Triac Trial II
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 2 years.
• 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.