Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional
Official Title: Effects of the Thyroid Hormone Analog Triac on the Neurocognitive Phenotype in Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter: The Triac Trial
Brief Summary:
Triac Trial II will assess the effects of Triac therapy on several neurocognitive end-points in patients with the Allan-Herndon-Dudley Syndrome (AHDS).
Patients with the AHDS suffer from severe psychomotor retardation. A mutation in the TH transporter protein Monocarboxylate transporter 8 (MCT8) results in reduced thyroid hormone (TH) levels in de brain, leading to an impaired neuronal differentiation. The severe neurological phenotype is accompanied by increased T3 levels in the blood, resulting in a variety of thyrotoxic symptoms such as tachycardia, increased metabolism, weight loss and a low muscle mass.
Currently, Triac Trial I (NCT02060474) investigates if Triac treatment in AHDS patients reduces the toxic effects of the high T3 levels. In addition, the safety of Triac administration in AHDS patients is assessed.
In Triac Trial II we will focus on the effects of Triac on the neurocognitive development.