Thyroid Hormone Analog Therapy in MCT8 Deficiency: Triac Trial Patients

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02060474
• Date of registration: 05/02/2014
• Prospective Registration: Yes
• Primary sponsor: Erasmus Medical Center
• Public title: Thyroid Hormone Analog Therapy in MCT8 Deficiency: Triac Trial Patients
• Scientific title: Thyroid Hormone Analog Therapy of Patients With Severe Psychomotor Retardation Caused by Mutations in the MCT8 Thyroid Hormone Transporter: The Triac Trial.
• Date of first enrolment: October 2014
• Target sample size: 46
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02060474
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Netherlands

Contacts

• Name: W.E. Visser, dr,
• Affiliation: Erasmus Medical Center

Key inclusion & exclusion criteria

Inclusion Criteria:

- clinically relevant mutation in the MCT8 gene, resulting in the clinical phenotype of
AHDS.

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);

- Known allergy to components in Triac tablets;

- Patients that have any contra-indication for Triac treatment.



Age minimum: N/A
Age maximum: 99 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Allan-Herndon-Dudley Syndrome

Intervention(s)

Drug: Triac

Primary Outcome(s)

serum T3 concentrations [Time Frame: Participants will be evaluated with an expected average of 2 weeks during months 1-3 of the trial. During months 4-12, participants will be evaluated with an expected average of 6 weeks. For statistical analysis baseline and month 12 will be compared]

serum reverse T3 concentrations [Time Frame: Participants will be evaluated with an expected average of 2 weeks during months 1-3 of the trial. During months 4-12 participants will be evaluated with an expected average of 6 weeks. For statistical analysis baseline and month 12 will be compared]

serum total T4 concentrations [Time Frame: Participants will be evaluated with an expected average of 2 weeks during months 1-3 of the trial. During months 4-12 participants will be evaluated with an expected average of 6 weeks. For statistical analysis baseline and month 12 will be compared]

serum free T4 concentrations [Time Frame: Participants will be evaluated with an expected average of 2 weeks during months 1-3 of the trial. During months 4-12 participants will be evaluated with an expected average of 6 weeks. For statistical analysis baseline and month 12 will be compared]

serum TSH concentrations [Time Frame: Participants will be evaluated with an expected average of 2 weeks during months 1-3 of the trial. During months 4-12 participants will be evaluated with an expected average of 6 weeks. For statistical analysis baseline and month 12 will be compared]

Secondary Outcome(s)

Body weight [Time Frame: baseline and month 12 will be compared]

serum sex-hormone binding globulin concentrations [Time Frame: baseline and month 12 will be compared]

Blood pressure [Time Frame: baseline and month 12 will be compared]

serum total cholesterol concentrations [Time Frame: baseline and month 12 will be compared]

serum creatine kinase concentrations [Time Frame: baseline and month 12 will be compared]

Heart rate [Time Frame: baseline and month 12 will be compared]

Secondary ID(s)

MCT8-2014-1

2014-000178-20

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

ZonMw: The Netherlands Organisation for Health Research and Development