NL-OMON53686尚未招募3 期
Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double-blind, Randomized, Placebo-controlled Study - reTRIACt
Rare Thyroid Therapeutics0 个研究点目标入组 5 人开始时间: 待定最近更新:
适应症
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 5
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 99（—）
入选标准
- •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
- •b) in the Screening Visit sample, or most recent standard of care sample prior
- •to screening, for participants who have never received and/or currently not
- •receiving tiratricol.
- •3. 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.
- •4. Signed and dated informed consent form from the parents or legal guardian.
排除标准
- •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
- •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
- •the investigational product (IP).
- •5. Participants with any contra-indication for treatment with tiratricol or any
- •excipients in the IP.
- •6. Participants using other T3 analogues, levothyroxine, or propylthiouracil.
- •In addition to these eligibility criteria, participants must meet further
- •criteria at the time
- •of randomization to enter the Randomized Treatment Period.
- •Randomization criteria:
- •1. Confirmation that the *Stable Dose Criterion* has been met.
- •2. Absence of any new or exacerbated medical or surgical condition that fulfils
- •Exclusion criterion #1.
- •3. Confirmation that participant is at least 4 years of age at the time of
- •randomization.
研究者
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