Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double-blind, Randomized, Placebo-controlled Study
EU CTIS ID: 2024-516124-34-00
What this study is testing
The primary objective is to evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total triiodothyronine (T3) concentrations, measured by liquid chromatography with tandem mass spectrometry (LC/MS/MS), as compared to continuing tiratricol treatment (tiratricol group), in males diagnosed with MCT8 deficiency and on a stable maintenance dose of tiratricol.
- Therapeutic confirmatory (Phase III)
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
- Participants will be enrolled in the present study if they meet the following eligibility criteria: 1. Male participants diagnosed with a pathogenic mutation in the MCT8 gene, confirmed with a genetic test. 2. Serum total T3 concentration above the ULN of the age-specific normal range: a) at the time of diagnosis (or the closest sample taken prior to first ever treatment with tiratricol) for participants who are currently treated with tiratricol (if serum total T3 concentration is not available, free T3 results from standard of care samples may be used). b) in the Screening Visit sample: i. For participants who have never received and/or are currently not receiving tiratricol. ii. For participants who stopped prohibited medications per exclusion criterion #6. 3. Participants will be aged 4 years or older at the time of randomization. Participants entering screening who are <4 years of age but expected to be aged 4 years at randomization should be discussed with the medical monitor. 4. Signed and dated informed consent form from the parents or legal guardian
You likely can't join if
- Participants will be excluded from the study if they meet any of the following criteria: 1. Major illness or recent major surgery unrelated to MCT8 deficiency (in the principal investigator’s judgement), defined as: • Conditions requiring repeated hospitalizations that are likely to confound ability to participate in the trial. • Major illness in the 3 months prior to the Screening Visit that is likely to confound the ability of the participant to participate fully within the trial and/or confound the assessment of serum total T3 and/or safety.• Major surgery within the 3 months prior to the Screening Visit or planned to take place during the study, including but not limited to major abdominal/thoracic/neurosurgical procedures. • Major/minor abdominal and/or maxillofacial surgery that may inhibit the administration and/or absorption of study drug. 2. Body weight <10 kg at the Screening Visit. 3. Patients who are participating, or intend to participate, in other therapeutic and/or interventional clinical studies during the study period. 4. History of allergic reactions to components of tiratricol or any excipients in the IP. 5. Participants with any contra-indication for treatment with tiratricol or any excipients in the IP. 6. Participants who have used other T3 analogues, levothyroxine, propylthiouracil, or other antithyroid medications within 6 weeks of screening
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
- 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, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom; 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.