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

Phase 1

• Conditions: Monocarboxylate Transporter 8 (MCT8) deficiency; Therapeutic area: Diseases [C] - Hormonal diseases [C19]

• Registration Number: EUCTR2019-003370-35-NL
• 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: 2020-03-17
• Last Update Posted: 5 January 2021

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 lactose)
6. Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.
7. Patients that have any contra-indication for tiratricol treatment as stated in the Investigators Brochure.

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: Evaluate the effects of tiratricol treatment on neurodevelopment in young MCT8 deficiency patients as measured by the Gross Motor Function Measure 88 (GMFM-88) and BSID-III Gross Motor Skill Domain.;Secondary Objective: 1) Evaluate the effect of tiratricol treatment on specific Motor Development milestones by individual item scores in the Gross Motor Function Measure.<br><br>2) Evaluate the effect of tiratricol treatment on neurodevelopment in young MCT8 deficient patients as measured by the Bayley Scales of Infant Development (BSID-III).<br><br>3) Evaluate the effect of tiratricol on clinical and biochemical thyrotoxic features (serum T3 concentrations, tissue specific markers of thyroid hormone action).;Primary end point(s): GMFM-88 total score and BSID Gross Motor Skill Domain compared to natural history scores from untreated patients.;Timepoint(s) of evaluation of this end point: After 24 months of treatment compared to baseline. An interim analysis will be done after 12 months treatment.

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): 1) GMFM-88 individual item score 10 and 24 compared to natural history scores from untreated patients. <br><br>2) Age equivalent (AE) score from the BSID-III compared to natural history AE scores from untreated patients. <br><br>3) Serum T3 (efficacy), peripheral thyroid hormone status (serum SHBG; serum creatine kinase, creatinine, blood pressure and body weight) (efficacy).<br>;Timepoint(s) of evaluation of this end point: After 24 months of treatment compared to baseline. An interim analysis will be done after 12 months treatment.