Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II (MCT8-2019-2)
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 13
- 试验地点
- 2
- 主要终点
- Part I of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at week 96 compared to natural history scores from the Triac Trial I study Part II of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at 3 years and 4 years respectively, compared to natural history scores from the Triac Trial I study.
研究概览
简要总结
Part I: Evaluate the effects of tiratricol treatment on neurodevelopment in young MCT8 deficiency patients, as measured by the Gross Motor Function Measure (GMFM-88) and Bayley Scales of Infant Development (BSID)-III Gross Motor Skill Domain. Part II: Evaluate the effects of long-term treatment (up to 4 years of total treatment) with tiratricol on neurodevelopment in young boys (≤30 months) with MCT8 deficiency, as measured by the Gross Motor Function Measure (GMFM)-88 and Bayley Scales of Infant Development (BSID)-III Gross Motor Skill Domain.
研究设计
- 研究类型
- Interventional
- 分配方式
- Not Applicable
- 主要目的
- Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II
- 盲法
- None
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
- •Signed and dated informed consent form from the parents or legal guardian.
- •Parents stated willingness to comply with all study procedures and availability for the duration of the study.
- •The participant should be aged between 0 and 30 months on the day of inclusion.
- •The participant should be male and have a pathogenic mutation in the MCT8 gene.
排除标准
- •Previous treatment with tiratricol.
- •Previous treatment with LT4 and/or PTU and/or other anti-thyroid medication for a period longer than three months. Patients previously treated with LT4 for a shorter period than 3 months may be included in the study (baseline visit) six weeks (or longer) after last dose of LT4 if two consecutive analyses show stable TFT*. Patients treated with PTU and/or other anti-thyroid medication for a shorter period than three months may be included in the study (baseline visit) six weeks (or longer) after last dose.
- •Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency.
- •Known allergic reactions to components of the IMP. Patients with galactose intolerance, Lapp lactase deficiency or malabsorption of glucose or galactose (the IMP contains lactose).
- •Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit *Stable TFT (T3, T4, fT4), determined as a maximal variation of 20%, should be demonstrated at two separate occasions at least two weeks apart, measured on the same platform.
结局指标
主要结局
Part I of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at week 96 compared to natural history scores from the Triac Trial I study Part II of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at 3 years and 4 years respectively, compared to natural history scores from the Triac Trial I study.
Part I of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at week 96 compared to natural history scores from the Triac Trial I study Part II of the study: GMFM-88 total score and BSID-III Gross Motor Skill Domain at 3 years and 4 years respectively, compared to natural history scores from the Triac Trial I study.
次要结局
- Part I and II:GMFM-88 individual item score 10 ("lifts head upright") and item score 24 ("sit on mat") at week 96, at 3 and 4 years respectively, compared to baseline; GMFM Domain B (Sitting) - summary score of all items 18-37 at week 96, at 3 and 4 years respectively, compared to baseline; Motor milestone responder analysis of Section 2 of the Hammersmith Infant Neurological Examination (HINE) at week 96, at 3 and 4 years respectively.
研究者
Clinical Trials
Scientific
Rare Thyroid Therapeutics International AB