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

• Register: EUCTR
• Last refreshed on: 20 July 2020
• Main ID: EUCTR2019-003370-35-GB
• Date of registration: 07/01/2020
• Prospective Registration: Yes
• Primary sponsor: Rare Thyroid Therapeutics International AB
• Public title: Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II
• Scientific title: Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II - Triac Trial II
• Date of first enrolment: 28/02/2020
• Target sample size: 22
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-003370-35
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no; Number of treatment arms in the trial: 1
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Czech Republic, France, Germany, Italy, Netherlands, United Kingdom, United States

Contacts

• Name: Medical Director
• Affiliation: Rare Thyroid Therapeutics International AB

• Name: Medical Director
• Affiliation: Rare Thyroid Therapeutics International AB

Key inclusion & exclusion criteria

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.



Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Monocarboxylate Transporter 8 (MCT8) deficiency
MedDRA version: 20.0 Level: PT Classification code 10078564 Term: Thyroid stimulating hormone deficiency System Organ Class: 10014698 - Endocrine disorders

Therapeutic area: Diseases [C] - Hormonal diseases [C19]

Intervention(s)

Product Name: Emcitate
Pharmaceutical Form: Tablet
INN or Proposed INN: TIRATRICOL
CAS Number: 51-24-1
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 100-

Product Name: Emcitate
Pharmaceutical Form: Tablet
INN or Proposed INN: TIRATRICOL
CAS Number: 51-24-1
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 350-

Primary Outcome(s)

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.

Primary end point(s): GMFM-88 total score and BSID Gross Motor Skill Domain compared to natural history scores from untreated patients.

Secondary Objective: 1) Evaluate the effect of tiratricol treatment on specific Motor Development milestones by individual item scores in the Gross Motor Function Measure.
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).
3) Evaluate the effect of tiratricol on clinical and biochemical thyrotoxic features (serum T3 concentrations, tissue specific markers of thyroid hormone action).

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(s)

Secondary end point(s): 1) GMFM-88 individual item score 10 and 24 compared to natural history scores from untreated patients.

2) Age equivalent (AE) score from the BSID-III compared to natural history AE scores from untreated patients.

3) Serum T3 (efficacy), peripheral thyroid hormone status (serum SHBG; serum creatine kinase, creatinine, blood pressure and body weight) (efficacy).

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 ID(s)

MCT8-2019-2

2019-003370-35-FR

NCT02396459

Source(s) of Monetary Support

Eurostars

Ethics review

Status: Approved
Approval date: 28/02/2020
Contact: