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 be treated for 24 months with tiratricol,
treatment effect on neurodevelopment impairment caused by hypothyroidism and the
thyrotoxicosis will be evaluated at an interim analysis after 12 months treatment and after
24 months treatment.
Phase:
Phase 2
Details
Lead Sponsor:
Erasmus Medical Center Rare Thyroid Therapeutics International AB
Collaborator:
Erasmus Medical Center
Treatments:
Chlorpheniramine, phenylpropanolamine drug combination Guaifenesin Phenylpropanolamine