Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Doubleblind, Randomized, Placebocontrolled Study (MCT8-2021-3 (ReTRIACt))

Conditions

Monocarboxylate Transporter 8 (MCT8) deficiency

Brief summary

Primary endpoint 1: Rate of change from baseline in serum total T3 during the 30-day double-blind Randomized Treatment Period. Primary endpoint 2: Proportion of participants who meet the rescue criterion (serum total T3 > ULN) during the 30-day double-blind Randomized Treatment Period.

Detailed description

Change in serum thyroid hormone variables (T3, T4, TSH, fT3 and fT4) from baseline to the end of Randomized Treatment Period, Change in serum thyroid hormone variables (T3, T4, TSH, fT3, and fT4) from screening to End of Study, Change in serum thyroid hormone variables (T3, T4, TSH, fT3, and fT4) from initiation of tiratricol administration at enrolment to the last measurement prior to randomization, Change in clinical endpoints: Heart rate (HR), systolic blood pressure (SBP) and rate pressure product (RPP) from baseline to the end of Randomized Treatment Period, Change in clinical endpoints: HR, SBP, RPP and body weight from screening to End of Study, Change in clinical endpoints: HR, SBP, RPP from initiation of tiratricol administration at screening to the last measurement prior to randomization

Related papers

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Interventions

DRUGTiratricol

DRUGMatching placebo (for participants allocated to the placebo group in the Randomized Treatment Period)

Eligibility

Sex/Gender

Male

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Primary endpoint 1: Rate of change from baseline in serum total T3 during the 30-day double-blind Randomized Treatment Period. Primary endpoint 2: Proportion of participants who meet the rescue criterion (serum total T3 > ULN) during the 30-day double-blind Randomized Treatment Period.	—

Secondary

Measure	Time frame
Change in serum thyroid hormone variables (T3, T4, TSH, fT3 and fT4) from baseline to the end of Randomized Treatment Period, Change in serum thyroid hormone variables (T3, T4, TSH, fT3, and fT4) from screening to End of Study, Change in serum thyroid hormone variables (T3, T4, TSH, fT3, and fT4) from initiation of tiratricol administration at enrolment to the last measurement prior to randomization, Change in clinical endpoints: Heart rate (HR), systolic blood pressure (SBP) and rate pressure product (RPP) from baseline to the end of Randomized Treatment Period, Change in clinical endpoints: HR, SBP, RPP and body weight from screening to End of Study, Change in clinical endpoints: HR, SBP, RPP from initiation of tiratricol administration at screening to the last measurement prior to randomization	—

Measure

Time frame

Change in serum thyroid hormone variables (T3, T4, TSH, fT3 and fT4) from baseline to the end of Randomized Treatment Period, Change in serum thyroid hormone variables (T3, T4, TSH, fT3, and fT4) from screening to End of Study, Change in serum thyroid hormone variables (T3, T4, TSH, fT3, and fT4) from initiation of tiratricol administration at enrolment to the last measurement prior to randomization, Change in clinical endpoints: Heart rate (HR), systolic blood pressure (SBP) and rate pressure product (RPP) from baseline to the end of Randomized Treatment Period, Change in clinical endpoints: HR, SBP, RPP and body weight from screening to End of Study, Change in clinical endpoints: HR, SBP, RPP from initiation of tiratricol administration at screening to the last measurement prior to randomization

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Countries

Netherlands

Outcome results

None listed

Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Doubleblind, Randomized, Placebocontrolled Study (MCT8-2021-3 (ReTRIACt))