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

Rare Thyroid Therapeutics International0 个研究点目标入组 18 人开始时间: 2021年5月24日最近更新: 2021年8月30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 have a pathogenic mutation in the MCT8 gene.
•Are the trial subjects under 18? yes
•Number of subjects for this age range:
•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.
•4. Treatment with LT4 within three months of baseline visit.
•5. Major illness or recent major surgery (within four weeks of baseline visit 1) unrelated to MCT8 deficiency.
•6. Known allergic reactions to components of the IMP.
•7. Treatment with another investigational drug or participation in other interventional trial within three months prior to baseline visit 1.
•8. Patients that have any contra-indication for tiratricol treatment as stated in the Investigators Brochure.

发起方

Rare Thyroid Therapeutics International