EICHORCondition: Monocarboxylate Transporter 8 Deficiency · Allan-Herndon-Dudley Syndrome · Sponsor: Rare Thyroid Therapeutics International AB
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.
This description comes directly from the study's public registry record.
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| Rare Disease Research, LLC | Kissimmee, Florida, United States | — |
| Rare Disease Research, LLC | Atlanta, Georgia, United States | — |
| SSM Health Cardinal Glennon Children's Hospital | St Louis, Missouri, United States | — |
| Rare Disease Research, LLC | Hillsborough, North Carolina, United States | — |
| Children's Hospital of Philadelphia | Philadelphia, Pennsylvania, United States | — |
| Tranquil Clinical and Research Consulting Services | Webster, Texas, United States | — |
| Erasmus MC | Rotterdam, Netherlands | — |
| Addenbrooke's Hospital | Cambridge, United Kingdom | — |
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Source record: clinicaltrials.gov/study/NCT05579327