MCT8-AHDS Foundation

Ongoing Medical Trials

Name of the trial: Rescue of Infants With Mct8 Deficiency Under Emergency Use Single Patient Expanded Access Treatment
Description: MCT8 deficiency (that is also known as Allan-Herndon-Dudley syndrome) is a rare X-linked inherited disorder of brain development that causes severe intellectual disability and problems with movement.
Location:  University of Miami, Miller School of Medicine, United States, Florida

Principal Investigator: Roy E Weiss, M.D.

Drug: Diiodothyropropionic acid  (DITPA)

Web page: https://clinicaltrials.gov/ct2/show/NCT04143295

 

Past Clinical Trials

Thyroid Hormone Analog Therapy of Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter: The Triac Trial.

Description:

The current trial will investigate if Triac treatment in AHDS patients:
1. reduces the toxic effects of the high T3 levels
2. restores the local TH deficiency in brain.

Primary Purpose: Treatment
Study Start Date: Oct 2014
Estimated completion date: May 2018
Location:  Erasmus Medical Center, Rotterdam
Principal Investigator: Dr. W.E. Wisser, Erasmus Medical Center
Recruitment Status : Active, not recruiting
Web page: https://clinicaltrials.gov/ct2/show/NCT02060474

 

Upcoming Trials

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 II

Description: The current Trial (Triac Trial II) will investigate if Triac treatment in AHDS patients improves the neurocognitive phenotype.
Primary Purpose:  Treatment
Estimated Study Start Date : June 2018
Estimated Study Completion Date : December 2022
Location:  Erasmus Medical Center, Rotterdam
Principal Investigator: Dr. W.E. Wisser, Erasmus Medical Center
Recruitment Status : Not yet recruiting
Web page: https://clinicaltrials.gov/ct2/show/NCT02396459