Study of Tiratricol Treatment Withdrawal in Males with MCT8 Deficiency (Monocarboxylate Transporter 8 Deficiency)

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What is this study about?

This study focuses on Monocarboxylate Transporter 8 (MCT8) deficiency, a rare genetic condition that affects how thyroid hormones move through the body. The research examines the effects of a medication called tiratricol, which is a thyroid hormone-like substance taken as tablets, in male patients who have been diagnosed with this condition.

The purpose of this research is to understand what happens when some patients stop taking tiratricol and receive a placebo instead, while others continue their regular tiratricol treatment. The study will specifically look at the levels of thyroid hormones in the blood and determine if patients who stop taking tiratricol need to restart the treatment.

During the study, participants will be randomly divided into two groups. One group will continue taking tiratricol tablets, while the other group will receive placebo tablets. The treatment period will last for 30 days, during which various measurements will be taken, including blood tests to check thyroid hormone levels, heart function monitoring, and general health assessments. The study includes regular check-ups to ensure participant safety throughout the treatment period.

1 Initial evaluation

Your diagnosis of MCT8 deficiency will be confirmed through genetic testing.

Blood tests will be performed to measure your thyroid hormone levels, particularly T3 hormone.

A medical examination will be conducted to ensure you meet the study requirements.

2 Treatment initiation

You will receive tiratricol tablets for oral use.

Your thyroid hormone levels will be monitored to establish the correct dosage.

Regular blood tests will track changes in your hormone levels.

3 Randomized treatment period

You will be randomly assigned to continue taking either tiratricol or placebo tablets.

This phase lasts 30 days.

Neither you nor your doctor will know which treatment you are receiving during this period.

4 Monitoring during treatment

Regular measurements will include:

Blood tests to check thyroid hormone levels

Heart monitoring through ECG and 24-hour blood pressure measurements

Physical examinations

Weight measurements

Blood tests for general health indicators

5 Safety monitoring

Any side effects or health changes will be recorded and monitored.

Additional treatment with tiratricol may be provided if your T3 levels become too high.

Regular checks of liver function, kidney function, and cholesterol levels will be performed.

Who Can Join the Study?

Must be male with confirmed MCT8 gene mutation (proven by genetic testing)
Must have elevated levels of T3 hormone (thyroid hormone) above the normal range either:
- At the time of diagnosis before starting tiratricol treatment, or
- During the screening visit (for those who have never taken tiratricol or have stopped other medications)
Must be at least 4 years old when starting the study (children who will turn 4 by the study start may be considered)
Must have parent or legal guardian consent through a signed and dated form

Who Cannot Join the Study?

Being female (only male participants are eligible)
Age under 18 years or over 65 years old
Not having a confirmed diagnosis of MCT8 deficiency
Not currently taking a stable dose of tiratricol (a thyroid hormone medication)
Having any serious medical conditions that could interfere with the study procedures
Being unable to comply with study procedures or follow-up visits
Having participated in another clinical trial within the past 30 days
Having any known allergies to tiratricol or similar medications
Having significant abnormalities in blood tests that could affect safety
Being unable to provide informed consent

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Evttpvp Uajwvckxnfgg Mnjmtju Caymlwk Rxxpzaphe (kgisxnd Mgo	Rotterdam	The Netherlands

Trial status

Country	Status	Recruitment Start
The Netherlands	Not recruiting	15.11.2022

Trial locations

Investigated drugs:

Tiratricol

Tiratricol is a medication that helps regulate thyroid hormone levels in patients with MCT8 deficiency, a rare genetic condition affecting thyroid hormone transport. This medication is particularly important for managing high T3 hormone levels in the blood of patients with this condition. It works by helping to normalize thyroid hormone function in the body. The medication helps improve symptoms associated with MCT8 deficiency, which is a condition that primarily affects males.

Monocarboxylate Transporter 8 (MCT8) Deficiency – A rare genetic condition that affects the body’s ability to transport thyroid hormones into cells. The condition primarily affects males and is caused by mutations in the MCT8 gene, which is responsible for moving thyroid hormones across cell membranes. People with MCT8 deficiency have abnormal thyroid hormone levels in their blood, with typically high T3 levels. This disorder affects brain development and function, leading to developmental delays and movement difficulties. The condition becomes apparent in early infancy, with affected individuals showing problems with muscle control and movement.

Trial ID:

2024-516124-34-00

Protocol code:

MCT8-2021-3

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study of Tiratricol Treatment Withdrawal in Males with MCT8 Deficiency (Monocarboxylate Transporter 8 Deficiency)

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?