Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Doubleblind, Randomized, Placebocontrolled Study (MCT8-2021-3 (ReTRIACt))
This important study, called ReTRIACt, is for adult men who have a rare condition called Monocarboxylate Transporter 8 (MCT8) deficiency. It's designed to understand what happens when a specific medication, Tiratricol, is withdrawn. Participants will be split into two groups – one continuing with Tiratricol and the other receiving a dummy treatment (placebo) for 30 days, without knowing which they are getting. Doctors will closely monitor their T3 hormone levels and general health to see how their bodies react to stopping the treatment. The main goal is to check how T3 levels change and if they go too high, which would trigger a return to the active medicine.
At a glance
What is this study about?
This study is looking into a rare inherited condition called Monocarboxylate Transporter 8 (MCT8) deficiency, which mainly affects boys and men. People with MCT8 deficiency have problems with how their bodies use thyroid hormones, particularly T3, which are really important for growth and development. This can lead to a range of health challenges.
The current study focuses on a treatment called Tiratricol. Doctors have been giving Tiratricol to some patients to help manage their hormone levels. This study wants to understand what happens when this treatment is stopped in men who are already taking it. Researchers need to see how their body’s T3 hormone levels change and if they remain stable or if they go outside of a healthy range. This information is very important for doctors to know the best way to manage MCT8 deficiency long-term.
Participants will be placed into two groups by chance, like flipping a coin. One group will continue with Tiratricol, and the other will switch to a 'dummy' treatment (placebo) for a short period, and neither the participants nor the doctors will know who is in which group until after the study period. This is called a 'double-blind' study and helps make sure the results are fair and accurate. They will carefully check the participants' T3 hormone levels and overall health during this time.
Key takeaways
- The study focuses on men with rare MCT8 deficiency.
- It investigates stopping a treatment called Tiratricol.
- Hormone levels, particularly T3, will be closely monitored.
- Patients will receive either Tiratricol or a dummy pill (placebo) for 30 days.
- The findings will help doctors better manage MCT8 deficiency.
Who may be eligible?
To be considered for this study, you must be an adult male, at least 18 years of age. There is no upper age limit, meaning older men are also able to take part.
You must have a confirmed diagnosis of Monocarboxylate Transporter 8 (MCT8) deficiency. The study is specifically for individuals with this particular rare condition.
Because this study is about stopping a treatment, you would also need to be currently receiving Tiratricol therapy for your MCT8 deficiency. Your doctors will be able to tell you if you meet all the necessary health criteria for safe participation.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Are you an adult male (aged 18 or older)?
- Do you have a diagnosis of MCT8 deficiency?
- Are you currently taking Tiratricol medication?
- Are you able to attend regular clinic visits?
What does participation involve?
If you decide to take part in this study, you would first have a screening visit to check if you meet all the requirements. If eligible, you would start a 30-day period where you would either continue your Tiratricol or switch to a placebo (a dummy medicine), without you or your doctor knowing which one you are taking. During this time, you would have several visits to the clinic so doctors can closely monitor your thyroid hormone levels (like T3 and T4) through blood tests. They will also check your general health, including your heart rate, blood pressure, and weight. The total duration of your participation, including follow-up, would be discussed with the study team.
Potential risks and benefits
Locations (1)
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Common questions
What is MCT8 deficiency?
It's a rare health condition, mostly affecting men, where the body has trouble using an important thyroid hormone called T3.
What is Tiratricol?
Tiratricol is a medicine that doctors use to help manage hormone levels in people with MCT8 deficiency.
Why is this study being done?
The study aims to understand what happens to T3 hormone levels and overall health when Tiratricol treatment is temporarily stopped in men with MCT8 deficiency.
Will I know if I'm getting Tiratricol or a placebo?
No, during the main part of the study, neither you nor your doctor will know if you are receiving Tiratricol or the placebo; this helps make the results fairer.
What are 'T3' and 'ULN'?
T3 is a type of thyroid hormone. ULN stands for 'Upper Limit of Normal', meaning the highest level considered healthy for T3.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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