Withdrawal of Tiratricol Treatment in Males With Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency)
Public ClinicalTrials.gov record NCT05579327. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Withdrawal of Tiratricol Treatment in Males With Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double-blind, Randomized, Placebo-controlled Study
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is a double-blind, randomized phase 3 multicenter placebo-controlled study in at least 16 evaluable male participants diagnosed with MCT8 deficiency. Male participants, from 4 years of age (at randomization) and having demonstrated stable maintenance treatment with tiratricol, will be randomized to receive placebo or tiratricol for 30 days or until reaching the rescue criterion (serum total triiodothyronine \[T3\] \> upper limit of normal \[ULN\] of the participant's normal range, for a sample collected during the 30-day Randomized Treatment Period). The research hypothesis to be tested is that, for participants in the placebo group, removal of tiratricol will lead to an increase of serum total T3 concentration, measured by liquid chromatography with tandem mass spectrometry (LC/MS/MS), above the ULN and requirement of rescue treatment with tiratricol, compared to those who continue to receive tiratricol.
Study identification
- NCT ID
- NCT05579327
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 20 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- 4 Years and older
- Sex
- Male
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jul 20, 2023
- Primary completion
- Jul 21, 2025
- Completion
- Sep 2, 2025
- Last update posted
- Apr 7, 2026
2023 – 2025
United States locations
- U.S. sites
- 6
- U.S. states
- 6
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Rare Disease Research, LLC | Kissimmee | Florida | 34746 | — |
| Rare Disease Research, LLC | Atlanta | Georgia | 30329 | — |
| SSM Health Cardinal Glennon Children's Hospital | St Louis | Missouri | 63104 | — |
| Rare Disease Research, LLC | Hillsborough | North Carolina | 27278 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
| Tranquil Clinical and Research Consulting Services | Webster | Texas | 77598 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 2 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05579327, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 7, 2026 · Synced Sep 10, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05579327 live on ClinicalTrials.gov.