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

Phase 2

Active, not recruiting

• Conditions: Monocarboxylate Transporter 8 (MCT8) deficiency

• Registration Number: 2024-516123-13-00
• Lead Sponsor: Rare Thyroid Therapeutics International AB

Brief Summary

Part I: Evaluate the effects of tiratricol treatment on neurodevelopment

in young MCT8 deficiency patients, as measured by the Gross Motor

Function Measure (GMFM-88) and Bayley Scales of Infant Development

(BSID)-III Gross Motor Skill Domain.

Part II: Evaluate the effects of long-term treatment (up to 4 years of

total treatment) with tiratricol on neurodevelopment in young boys (≤30

months) with MCT8 deficiency, as measured by the Gross Motor Function

Measure (GMFM)-88 and Bayley Scales of Infant Development (BSID)-III

Gross Motor Skill Domain.

Detailed Description

Not available

Study Timeline

• Study First Posted: 2024-08-26
• Last Update Posted: 2025-06-06

Recruitment & Eligibility

• Status: Ongoing, recruitment ended
• Sex: Male
• Target Recruitment: 13

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.

Exclusion Criteria

1. Previous treatment with tiratricol. 2. Previous treatment with LT4 and/or PTU and/or other anti-thyroid medication for a period longer than three months. Patients previously treated with LT4 for a shorter period than 3 months may be included in the study (baseline visit) six weeks (or longer) after last dose of LT4 if two consecutive analyses show stable TFT*. Patients treated with PTU and/or other anti-thyroid medication for a shorter period than three months may be included in the study (baseline visit) six weeks (or longer) after last dose. 3. Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency. 4. Known allergic reactions to components of the IMP. Patients with galactose intolerance, Lapp lactase deficiency or malabsorption of glucose or galactose (the IMP contains lactose). 5. Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit *Stable TFT (T3, T4, fT4), determined as a maximal variation of 20%, should be demonstrated at two separate occasions at least two weeks apart, measured on the same platform.

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Part I of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at week 96 compared to natural history scores from the Triac Trial I study Part II of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at 3 years and 4 years respectively, compared to natural history scores from the Triac Trial I study.		Part I of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at week 96 compared to natural history scores from the Triac Trial I study Part II of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at 3 years and 4 years respectively, compared to natural history scores from the Triac Trial I study.

Secondary Outcome Measures

Name	Time	Method
Part I and II:GMFM-88 individual item score 10 ("lifts head upright") and item score 24 ("sit on mat") at week 96, at 3 and 4 years respectively, compared to baseline; GMFM Domain B (Sitting) - summary score of all items 18-37 at week 96, at 3 and 4 years respectively, compared to baseline; Motor milestone responder analysis of Section 2 of the Hammersmith Infant Neurological Examination (HINE) at week 96, at 3 and 4 years respectively.		Part I and II:GMFM-88 individual item score 10 ("lifts head upright") and item score 24 ("sit on mat") at week 96, at 3 and 4 years respectively, compared to baseline; GMFM Domain B (Sitting) - summary score of all items 18-37 at week 96, at 3 and 4 years respectively, compared to baseline; Motor milestone responder analysis of Section 2 of the Hammersmith Infant Neurological Examination (HINE) at week 96, at 3 and 4 years respectively.

Trial Locations

Locations (2)

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC); 🇳🇱 Rotterdam, Netherlands

Fakultni Nemocnice V Motole; 🇨🇿 Prague, Czechia

Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)

🇳🇱Rotterdam, Netherlands

Edward Visser

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