Monocarboxylate Transporter 8 (MCT8) deficiency
Conditions
Interventions
Product Name: Emcitate
Pharmaceutical Form: Tablet
INN or Proposed INN: TIRATRICOL
CAS Number: 51-24-1
Concentration unit: µg microgram(s)
Concentration type: equal
Concentration number: 350-
Pharmace
Sponsors
Rare Thyroid Therapeutics International AB
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion 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 =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion 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 investigational product (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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: 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), and the requirement for rescue treatment with tiratricol as compared to continuing tiratricol treatment (tiratricol group), in males diagnosed with MCT8 deficiency and on a stable maintenance dose of tiratricol;Secondary Objective: 1. To evaluate the safety and tolerability of tiratricol treatment 2. To evaluate the effect of tiratricol treatment upon serum thyroid hormone measurements, sex hormone binding globulin (SHBG), and cardiovascular measurements 3. To evaluate the serum concentrations of tiratricol ;Primary end point(s): Proportion of participants who meet the rescue criterion (serum total T3 > ULN) from samples obtained during the 30 day double-blind Randomized Treatment Period;Timepoint(s) of evaluation of this end point: end of Randomized Treatment period | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Change in serum thyroid hormone variables (T3, T4, TSH, fT3, and fT4) from baseline (start of the Randomized Treatment Period) to the end of Randomized Treatment Period (completion or rescue) • Change in serum thyroid hormone variables (T3, T4, TSH, fT3, and fT4) from initiation of tiratricol administration at screening to the last measurement prior to randomization (Cohort B only) • Change in the cardiovascular variables from extended ECG assessments, 24 hour ABPM, and heart rate assessments from the last measurement prior to the start of the Randomized Treatment Period to the end of the Randomized Treatment Period (completion or rescue) • Serum concentrations of tiratricol • Change in serum SHBG from baseline (start of the Randomized Treatment Period) to the end of the Randomized Treatment Period (completion or rescue) • Time (days) from randomization to the time when the rescue criterion is met or the time of completion of the Randomized Treatment Period (whichever comes first) ;Timepoint(s) of evaluation of this end point: end of Randomized Treatment period | — |
Countries
Netherlands, United Kingdom, United States
Contacts
Public ContactMedical Director
Rare Thyroid Therapeutics International AB
Outcome results
None listed