Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)
Responsive
Rotterdam, The Netherlands
Not recruitingRare disease
Study of tiratricol treatment in children with MCT8 deficiency (Monocarboxylate Transporter 8 deficiency) to improve motor development
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What is this trial about?
A plain-language summary of the goals, design and what participants do
This clinical trial focuses on Monocarboxylate Transporter 8 (MCT8) deficiency, a rare condition that affects the body's ability to properly transport thyroid hormones. The study examines the effectiveness of tiratricol, a thyroid hormone-like medication that comes in tablet form and is taken by mouth.
The study aims to determine how well tiratricol helps improve movement and development in young boys with MCT8 deficiency. The treatment period is divided into two parts, with participants receiving the medication for up to 4 years. During this time, doctors will monitor how the children's motor skills and overall development progress.
The medication being tested, tiratricol, is classified as a thyromimetic drug, which means it works similarly to natural thyroid hormones in the body. The study focuses specifically on young male patients who are diagnosed with MCT8 deficiency through genetic testing and are under the age of 30 months when starting the treatment.
The research process
The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.
Who can join the trial?
6 criteria
- Must be a male child aged between 0 and 30 months at the time of joining the study
- Must have a confirmed genetic mutation in the MCT8 gene (a gene that helps transport thyroid hormones in the body)
- Parents or legal guardian must sign and complete an informed consent form agreeing to participate in the study
- Parents must be willing and able to:
- Follow all required study procedures
- Attend all scheduled study visits
- Remain available throughout the entire study duration
Who cannot join the trial?
11 criteria
- Male patients over 30 months of age cannot participate in this study
- Female patients are not eligible for participation
- Patients who do not have confirmed MCT8 deficiency diagnosis through genetic testing
- Patients who are currently participating in other clinical trials
- Patients with severe allergic reactions to thyroid hormones or similar medications
- Patients with uncontrolled heart problems
- Patients with severe kidney or liver problems
- Patients who are taking medications that could interact with the study drug
- Patients who cannot complete required study visits and assessments
- Patients whose caregivers cannot provide informed consent
- Patients who have received other experimental treatments in the past 30 days
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Fakultni nemocnice Motol a Homolka
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Prague, Czechia
Trial locations
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Investigated drugs
Tiratricol is a medication used to treat children with a rare genetic condition called MCT8 deficiency, which affects thyroid hormone transport. This medication is designed to help improve neurodevelopment, particularly focusing on motor skills and movement abilities in young patients. It works by mimicking thyroid hormones and can reach areas of the body where natural thyroid hormones cannot be properly transported due to the genetic defect.
What is already known about the treatment
Tiratricol – A thyroid hormone analog administered orally that is being investigated for the treatment of Monocarboxylate Transporter 8 (MCT8) deficiency, a rare genetic condition affecting thyroid hormone transport. The medication works by mimicking thyroid hormones and can cross cell membranes without requiring the MCT8 transporter, which is defective in patients with this condition. Tiratricol belongs to the class of thyroid hormone derivatives and is being studied particularly for its effects on neurodevelopment in young patients, with clinical trials focusing on improving motor function and developmental outcomes in children with MCT8 deficiency.
Investigated diseases
Monocarboxylate Transporter 8 (MCT8) Deficiency - A rare genetic condition that affects the body's ability to transport thyroid hormones into cells, particularly in the brain. The condition primarily affects males and becomes apparent in early infancy or early childhood. It is characterized by problems with movement, muscle control, and developmental delays. Children with MCT8 deficiency typically have difficulty with physical activities like holding up their head, sitting, crawling, and walking. The condition also impacts intellectual development and speech abilities. This disorder is caused by mutations in the SLC16A2 gene, which provides instructions for making the MCT8 protein.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IITrial ID2024-516123-13-00Protocol codeMCT8-2019-2Estimated enrolment22 patientsSponsorRare Thyroid Therapeutics International AB
sourced from the EU Clinical Trials Register and site verification
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