ISSN 1662-4009 (online)

ey0017.3-11 | Clinical trials for thyroid disease | ESPEYB17

3.11. Thyroid hormone and folinic acid in young children with Down syndrome: the phase 3 ACTHYF trial

C Mircher , S Sacco , C Bouis , J Gallard , A Pichot , E Le Galloudec , Cieuta , I Marey , O Greiner-Mahler , N Dorison , A Gambarini , S Stora , S Durand , M Polak , A Baruchel , E Schlumberger , J Dewailly , A Azar-Kolakez , RM Gueant-Rodriguez , JL Gueant , D Borderie , D Bonnefont-Rousselot , E Blondiaux , A Ravel , FG Sturtz

To read the full abstract: Genet Med. 2020;22:44–52.This single center, randomized, double-blind, placebo-controlled phase 3 study investigated the effects of levothyroxine, folic acid, or both in combination over 12 months on global development in 143 infants with Down syndrome (DS). Over the last decades, treatment of DS associated co-morbidities has improved their life-expectancy. However, a therapy to improve mental development is still...