Therapy of MCT8 patients with the thyroid hormone analog Triac.

Phase 1

• Conditions: 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]

• Registration Number: EUCTR2014-000178-20-CZ
• Lead Sponsor: Erasmus Medical Centre

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-06-23
• Last Update Posted: 9 January 2017

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 40

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: 1
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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)

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified