Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II
- Conditions
- Monocarboxylate Transporter 8 (MCT8) deficiencyMedDRA version: 20.0Level: PTClassification code 10078564Term: Thyroid stimulating hormone deficiencySystem Organ Class: 10014698 - Endocrine disordersTherapeutic area: Diseases [C] - Hormonal diseases [C19]
- Registration Number
- EUCTR2019-003370-35-IT
- Lead Sponsor
- Rare Thyroid Therapeutics International
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Male
- Target Recruitment
- 18
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 have a pathogenic mutation in the MCT8 gene.
Are the trial subjects under 18? yes
Number of subjects for this age range:
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
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.
4. Treatment with LT4 within three months of baseline visit.
5. Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency.
6. Known allergic reactions to components of the IMP.
7. Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.
8. 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: To evaluate the effects of tiratricol treatment on neurodevelopment in young MCT8 deficiency patients as measured by the Gross Motor Function Measure 88 (GMFM-88).;Secondary Objective: 1) To evaluate the effect of tiratricol treatment on specific Motor Development milestones by individual item scores in the Gross Motor Function Measure test. <br><br>2) To 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 score compared to natural history GMFM-88 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) Gross Motor Function Measure test (GMFM-88) individual item score 10 and 24 compared to natural history scores from untreated patients. <br>2) Age equivalent (AE) score from the BSID compared to natural history AE scores from untreated patients. <br>3) Serum T3 (efficacy), peripheral thyroid hormone TH 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.