Therapy of MCT8 patients with the thyroid hormone analog Triac.

• Register: EUCTR
• Last refreshed on: 27 January 2025
• Main ID: EUCTR2014-000178-20-IT
• Date of registration: 16/10/2015
• Prospective Registration: Yes
• Primary sponsor: Erasmus Medical Centre
• Public title: Therapy of MCT8 patients with the thyroid hormone analog Triac.
• Scientific title: Thyroid hormone analog therapy of patients with severe psychomotor retardation caused by mutations in the MCT8 thyroid hormone transporter: The Triac Trial. - Triac Trial in MCT8 patients
• Date of first enrolment: 01/02/2016
• Target sample size: 40
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-000178-20
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Czech Republic, France, Germany, Italy

Contacts

• Name: Information Triac Trial
• Affiliation: Erasmus Medical Centre

• Name: Information Triac Trial
• Affiliation: Erasmus Medical Centre

Key inclusion & exclusion criteria

Inclusion criteria:
clinical relevant mutation in the MCT8 gene leading to the clinical phenotype of AHDS
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 3
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion criteria:
- Major illness or recent major surgery (within 4 weeks) unrelated to AHDS;
- Patients who are participating in ongoing RCTs of therapeutic interventions (including clinical trials of investigational medicinal products);
- Patients that have any major contra-indication for Triac treatment (severe cardiac decompensation (NYHA 4), coronary insufficiency, severe cardiac arrhytmias, Galactose intolerance, the Lapp lactose deficiency or glucose-galactose malabsorption).


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

This therapuetical trial will be conducted in patient with the Allan-Herndon-Dudley Syndrome (AHDS), casued by mutations in the thyroid hormone transporter MCT8. This results in the characteristic clinical phenotype of severe psychomotor retardation due to local hypothyroidism in the brain, in combination with high serum T3 and high normal serum TSH levels that lead to local hyperthyroidism in tissues that do not dependent on MCT8, resulting in tachycardia, low body weight and muscle wasting.

Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Intervention(s)

Trade Name: Téatrois (Tiratricol)
Product Name: Téatrois
Pharmaceutical Form: Tablet

Primary Outcome(s)

Main Objective: (1)To determine the effect of Triac treatment on serum T3 and other TH levels.
(2)To determine the effect of Triac on the toxic effects of the hyperthyroid state in peripheral tissues.

Timepoint(s) of evaluation of this end point: These primary endpoints will be evaluated at baseline (t=0) and after 1 year of Triac treatment.
To optimize titration of the Triac dose and prevent overtreatment, primary end-points will be evaluated with an expected average of 2 weeks during months 1-3 of the trial. During months 4-12 these parameters will be evaluated with an expected average of 6 weeks.

Primary end point(s): 1)Serum TSH, T4, vrij T4, T3, rT3 and Triac levels

Secondary Objective: (1)To determine the effects of Triac treatment on the neurological phenotype.
(2)The registration of adverse event.

Secondary Outcome(s)

Secondary end point(s): 1) Body weight, blood pressure and heart rate.
2) Serum levels of markers that reflect peripheral thyroid hormone action: serum steroid hormone binding globuline (SHBG) and lipids (liver), serum beta Ctx and alkalisch phosphatase (bone) and serum kreatine kinase (muscle).
3) Motor function, using the Gross Motor Function Measure.
4) Cognitive function using the Bayley Scales of Infant Development III, or Wechsler Preschool and Primary Scale of Intelligence II.
5) Adaptive behavior measured by the Vineland adaptive behavior scale.
6) Basal Metabolic Rate (BMR) using Doubly labeled Water (DLW).
7) The frequency and nature of adverse events.

Timepoint(s) of evaluation of this end point: The secundary end points 1-3 will will be evaluated at baseline (t=0) and after 1 year of Triac treatment.
To optimize titration of the Triac dose and prevent overtreatment, these end points will also be evaluated with an expected average of 2 weeks during months 1-3 of the trial. During months 4-12 these parameters will be evaluated with an expected average of 6 weeks.
End points 3), 4) and 5) will be evaluated at baseline and after 1 year of Triac treatment.
Point 6) will be evaluated during the whole study period.

Secondary ID(s)

2014-000178-20-BE

NCT02060474

MCT8-2014-1

Source(s) of Monetary Support

ZonMw

Ethics review

Status: Approved
Approval date: 28/10/2015
Contact: