Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II

Phase 1

• Conditions: Monocarboxylate Transporter 8 (MCT8) deficiency; MedDRA version: 20.0Level: PTClassification code 10078564Term: Thyroid stimulating hormone deficiencySystem Organ Class: 10014698 - Endocrine disorders; Therapeutic area: Diseases [C] - Hormonal diseases [C19]

• Registration Number: EUCTR2019-003370-35-GB
• Lead Sponsor: Rare Thyroid Therapeutics International AB

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-01-07
• Last Update Posted: 20 July 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: 22

Inclusion Criteria

1. Signed and dated informed consent form from the parents or legal guardian.
2. Parents stated willingness to comply with all study procedures and availability for the duration of the study.
3. The participant should be aged between 0 and 30 months on the day of inclusion.
4. The participant should be male and have a pathogenic mutation in the MCT8 gene.

Are the trial subjects under 18? yes
Number of subjects for this age range: 22
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

1. Previous treatment with tiratricol.
2. Previous treatment with a combination of Propylthiouracil (PTU) and Levothyroxine (LT4).
3. Previous treatment with LT4 for a longer period than three months. Treatment with LT4 within three months of baseline visit.
4. Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency.
5. Known allergic reactions to components of the IMP. Patients with galactose intolerance, Lapp lactose deficiency or malabsorption of glucose or galactose (the IMP contains lectose).
6. Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.

Study & Design

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