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Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II
EU CTIS ID: 2024-516123-13-00
What this study is testing
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.
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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.
You likely can't join if
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.