Tiratricol MCT8 Withdrawal Study
Research type
Research Study
Full title
Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double-blind, Randomized, Placebo-controlled Study
IRAS ID
1007165
Contact name
Kristina Sjoblom
Contact email
Sponsor organisation
Rare Thyroid Therapeutics
Eudract number
2022-001478-78
Clinicaltrials.gov Identifier
Research summary
MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome (AHDS), is a chronic and severely debilitating disease. It is a rare genetic disorder that occurs exclusively in males. Tiratricol is thyroid hormone metabolite. This means that it is usually naturally produced in the body during the breakdown of the natural thyroid hormone known as triiodothyronine, also called “T3”. In MCT8 deficiency, T3 levels are too
high. High T3 levels in the body will result in “thyrotoxicosis” which results in symptoms and signs such as a racing heart, rapid breathing, sweating, agitation, and weight loss. The consequences of this chronic thyroid hormone overexposure is believed to contribute to increased mortality. Tiratricol is intended to reduce the amount of T3 in the body to normal levels. By reducing the level of T3 in the body this is likely to reduce the symptoms and signs of “thyrotoxicosis.” In this study, tiratricol will be compared to a placebo, which is a tablet that looks exactly like tiratricol but does not contain any active ingredient. This study includes a Screening and a Follow-up Period, in which the child will receive tiratricol. There is a 30-day Treatment Period, during which the child will receive either tiratricol or placebo. This study will be randomized and neither the doctor or the patient will know whether they have been given Tiratricol or placebo and double blind so neither patient or doctor will know which treatment is given. This study is being run by a company called Rare Thyroid Therapeutics.REC name
East of England - Cambridge South Research Ethics Committee
REC reference
23/EE/0047
Date of REC Opinion
30 Aug 2023
REC opinion
Further Information Favourable Opinion