Tiratricol treatment of children with Monocarboxylate Transporter 8 deficiency: Triac Trial II - Triac Trial II

Rare Thyroid Therapeutics International AB0 个研究点目标入组 22 人开始时间: 2020年1月13日最近更新: 2020年8月3日

适应症

试验速览

暂无简介。

•1. Signed and dated informed consent form from the parents or legal guardian.
•2. Parents stated willingness to comply with all study procedures and availability for the duration of the study.
•3. The participant should be aged between 0 and 30 months on the day of inclusion.
•4. The participant should be male and have a pathogenic mutation in the MCT8 gene.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 22
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. Previous treatment with tiratricol.
•2. Previous treatment with a combination of Propylthiouracil (PTU) and Levothyroxine (LT4).
•3. Previous treatment with LT4 for a longer period than three months. Treatment with LT4 within three months of baseline visit.
•4. Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency.
•5. Known allergic reactions to components of the IMP. Patients with Lapp lactase deficiency or malabsorption of
•glucose or galactose (the IMP contains lactose).
•6.Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.

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