Triac Trial II in MCT8 Deficiency Patients

NCT02396459 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: MCT8-2019-2
• Study Type: interventional
• Target: 22
• Locations: 4 countries
• Sites: 5

Brief Summary

This study will investigate the effect of treatment with tiratricol (also called Triac) in young boys (≤30 months) with MCT8 deficiency (also called the Allan-Herndon-Dudley syndrome (AHDS)). The hypothesis tested is that treatment with tiratricol will have a beneficial effect on the hypothyroid state in the brain as well as the hyperthyroid state in peripheral organs and tissues in these patients. Patients will initially be treated for 96 weeks with tiratricol, treatment effect on neurodevelopment impairment caused by hypothyroidism and peripheral thyrotoxicosis will be evaluated after 96 weeks treatment. Patients will be offered to continue on treatment for an additional 3 years.

Conditions

Allan-Herndon-Dudley Syndrome

Keywords

Triac; MCT8; MCT8 deficiency; Allan-Herndon-Dudley Syndrome

Outcome Measures

Primary Outcomes (2)

• Gross Motor Function Measure 88 (GMFM 88) total score

  To evaluate the effects of tiratricol on neurodevelopment in young MCT8 deficiency patients, measured by the Gross Motor Function Measure (GMFM)-88 assessment. Potential result values range from 0 to 100%, the latter being representative for a 4-year old healthy child. A high score is equivalent to better/more neurodevelopment and is therefore a better outcome than a low score.

  96 weeks, Year 3, Year 4 and Year 5

• Bayley Scales of Infant Development III Gross Motor Skill Domain score

  To evaluate the effect of tiratricol treatment on neurodevelopment measured by the Bayley Scales of Infant Development (BSID-III) Gross Motor Skill Domain score. Potential total raw scores range from 0-72, and can be age-adjusted before analysis. A high score is equivalent to better/more neurodevelopment and is therefore a better outcome than a low score

  96 weeks, Year 3, Year 4 and Year 5

Secondary Outcomes (8)

• GMFM-88 individual item score 10 and 24.

  96 weeks, Year 3, Year 4 and Year 5

• Bayley Scales of Infant Development III score.

  96 weeks, Year 3, Year 4 and Year 5

• Serum T3 concentrations

  96 weeks, Year 3, Year 4 and Year 5

• Tissue-specific markers of thyroid state: serum sex-hormone binding globulin concentrations for liver

  96 weeks, Year 3, Year 4 and Year 5

• Tissue-specific markers of thyroid state: serum creatine kinase concentrations for muscles

  96 weeks, Year 3, Year 4 and Year 5

Other Outcomes (30)

• Patient Quality of Life by Infant Toddler Quality of Life Questionnaire (ITQoL)

  96 weeks and Year 5

• Parent Quality of Life by Parenting Stress index (PSI-4 SF)

  96 weeks and Year 5

• Evaluate the effect of tiratricol treatment on neurological symptoms (Hammersmith Infant Neurological Exam, HINE)

  96 weeks, Year 3, Year 4 and Year 5

Study Arms (1)

MCT8 deficient patients

EXPERIMENTAL

Tiratricol (Triac) treatment

Drug: Tiratricol

Interventions

Tiratricol DRUG

Tiratricol, individually titrated dose

Also known as: Triac, Teatrois

MCT8 deficient patients

Eligibility Criteria

• Age: Up to 30 Months
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Signed and dated informed consent form from the parents or legal guardian.
• Parents stated willingness to comply with all study procedures and availability for the duration of the study.
• The participant should be male and have a pathogenic mutation in the MCT8 gene.

You may not qualify if:

• Previous treatment with tiratricol.
• 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.
• Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency.
• 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).
• Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.

Sponsors & Collaborators

• Erasmus Medical Center: collaborator
• Rare Thyroid Therapeutics International AB: lead

Study Sites (5)

Oregon Health & Science University (OHSU) Doernbecher Childrens Hospital

Portland, Oregon, 97239, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Charles University and Motol University Hospital; The department of peadiatrics of the 2nd faculty of medicine

Prague, 15006, Czechia

Location

Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie

Berlin, 13353, Germany

Location

Erasmus MC

Rotterdam, 3015 GD, Netherlands

Location

MeSH Terms

Conditions

Allan-Herndon-Dudley syndrome

Interventions

3,3',5-triiodothyroacetic acid

Study Officials

• W.E. Visser, MD, PhD

  Erasmus Medical Center

  PRINCIPAL INVESTIGATOR

• Kristina Sjöblom Nygren, MD

  Rare Thyroid Therapeutics International AB

  STUDY DIRECTOR

• Lindsey Nicol

  Oregon Health& Science University (OHSU) Doernbecher Childrens Hospital

  PRINCIPAL INVESTIGATOR

• Jan Lebl

  Charles University and Motol University Hospital

  PRINCIPAL INVESTIGATOR

• Heiko Krude

  Charité - Universitätsmedizin Berlin Institut fur experimental paediatrische endokrinologie

  PRINCIPAL INVESTIGATOR

• Andrew Bauer, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 7, 2015
• First Posted: March 24, 2015
• Study Start: December 7, 2020
• Primary Completion (Estimated): July 18, 2027
• Study Completion (Estimated): August 18, 2027
• Last Updated: February 20, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (2); NL (1); DE (1); CZ (1)