|
|
|
Originator Org.- |
|
Inactive Indication- |
Drug Highest PhaseApproved |
First Approval Ctry. / Loc.- |
First Approval Date- |
Withdrawal of Tiratricol Treatment in Males With Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double-blind, Randomized, Placebo-controlled Study
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 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.
Phase I Trial: Quotient Code QSC300535
Caregiver-reported Economic Burden and Quality of Life Impact of Monocarboxylate Transporter 8 (MCT8) Deficiency
Caregivers face many responsibilities outside of their role as a friend or parent, which can lead to emotional, financial, social, and professional challenges. To better understand the impact of MCT8 deficiency on caregivers, Egetis Therapeutics are conducting an online survey for adult caregivers of persons living with the MCT8 deficiency.
100 Clinical Results associated with Rare Thyroid Therapeutics International AB
0 Patents (Medical) associated with Rare Thyroid Therapeutics International AB
100 Deals associated with Rare Thyroid Therapeutics International AB
100 Translational Medicine associated with Rare Thyroid Therapeutics International AB