Triac Trial II
Research type
Research Study
Full title
Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II
IRAS ID
275579
Contact name
Krishna Chatterjee
Contact email
Sponsor organisation
Rare Thyroid Therapeutics International AB
Eudract number
2019-003370-35
Clinicaltrials.gov Identifier
Clinicaltrials.gov Identifier
142377, Pre-IND number
Duration of Study in the UK
2 years, 11 months, 31 days
Research summary
This study looks at the effects of a thyroid hormone called tiratricol in boys aged under 30 months with a diagnosis of MCT8 deficiency (Monocarboxylate Transporter 8 deficiency, or Allan-Herndon-Dudley Syndrome (AHDS)), a disease caused by a mutation (abnormality) in the MCT8 protein.
Thyroid hormones are important for the development of the brain and many other tissues in the human body. Hormones need to enter the cells of these tissues via “gates/channels” (thyroid hormone transporter proteins). One of these gates is called MCT8. The brain depends on MCT8 to allow thyroid hormones to enter. If MCT8 is not working properly, lower levels of thyroid hormone will enter the brain, resulting in developmental abnormalities. This is the cause of the severe developmental and neurological problems which are seen in patients with MCT8 deficiency.
At the same time, other body tissues, such as the muscles and liver, do not depend on MCT8 to allow thyroid hormones to enter their cells. In patients with MCT8 deficiency, the blood level of active thyroid hormones in these organs is too high, which results in adverse effects such as muscle wasting and a low body weight.
The purpose of this study is to see whether treatment with tiratricol could help increase the levels of thyroid hormone in the brain and normalise the level of thyroid hormone in the rest of the body.
In this study patients will be given a daily dose of tiratricol (or Triac)orally or via a PEG tube for a period of 2 years. During a dose titration period in the beginning of the study the dose level of tiraticol will be gradually increased until the thyroid hormone levels in the blood reach the target level, or until side-effects occur. The patient will then be treated with this individually titrated dose for the rest of the study period.REC name
East Midlands - Derby Research Ethics Committee
REC reference
20/EM/0035
Date of REC Opinion
28 Feb 2020
REC opinion
Further Information Favourable Opinion