2024-516124-34-00招募中3 期
Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Doubleblind, Randomized, Placebocontrolled Study (MCT8-2021-3 (ReTRIACt))
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- The primary efficacy endpoint is the proportion of participants who meet the rescue criterion (serum total T3 > ULN) from samples obtained during the 30-day double-blind Randomized Treatment Period.
研究概览
简要总结
The primary objective is to evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total 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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 主要目的
- Withdrawal of Tiratricol treatment of children with Monocarboxylate Transporter 8deficiency:ReTRIACT
- 盲法
- Double (Investigator, Subject, Monitor, Carer)
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •Participants will be enrolled in the present study if they meet the following eligibility criteria:
- •Male participants diagnosed with a pathogenic mutation in the MCT8 gene, confirmed with a genetic test.
- •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 #
- •Participants will be aged 4 years or older at the time of randomization. Participants entering screening who are <4 years of age but expected to be aged 4 years at randomization should be discussed with the medical monitor.
- •Signed and dated informed consent form from the parents or legal guardian
排除标准
- •Participants will be excluded from the study if they meet any of the following criteria:
- •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.
- •Body weight <10 kg at the Screening Visit.
- •Patients who are participating, or intend to participate, in other therapeutic and/or interventional clinical studies during the study period.
- •History of allergic reactions to components of tiratricol or any excipients in the IP.
- •Participants with any contra-indication for treatment with tiratricol or any excipients in the IP.
- •Participants who have used other T3 analogues, levothyroxine, propylthiouracil, or other antithyroid medications within 6 weeks of screening
结局指标
主要结局
The primary efficacy endpoint is the proportion of participants who meet the rescue criterion (serum total T3 > ULN) from samples obtained during the 30-day double-blind Randomized Treatment Period.
The primary efficacy endpoint is the proportion of participants who meet the rescue criterion (serum total T3 > ULN) from samples obtained during the 30-day double-blind Randomized Treatment Period.
次要结局
- 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)
- Safety evaluation: Adverse events, Safety laboratory variables (full blood count, serum renal and liver biomarkers, total and LDL cholesterol), Standard vital signs variables (temperature, respiratory rate, pulse rate, and blood pressure), 24 hour ambulatory blood pressure monitoring (ABPM), Weight, ECG evaluations, Physical examinations
研究者
Clinical Trials
Scientific
Rare Thyroid Therapeutics International AB
研究点 (1)
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