ACTRN12611000472976招募中1 期
A multi-centre, open-label study to determine the safety, tolerability and pharmacodynamics of orally administered 3,5-diiodothyropropionic acid (DITPA) in patients aged 3 to 12 with Allan-Herndon-Dudley Syndrome (AHDS)
Zarion Pharmaceuticals0 个研究点目标入组 10 人开始时间: 2011年5月6日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 10
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- Non-randomised trial
- 主要目的
- Treatment
- 盲法
- Open (masking not used)
入排标准
- 年龄范围
- Years 至 12 Years（—）
- 性别
- All
入选标准
- •1. Proven mutation of the SLC16A2/MCT8 gene, with a presumptive diagnosis of MCT8 Deficiency made based on elevated fT3, decrease of serum rT3 concentrations, low fT4 in the blood.
- •2. Medically stable according to the treating physician.
- •3. Informed parental consent.
- •4. Aged between 0 and 12 years.
排除标准
- •1. Other forms of hypo- or hyperthyroidism.
- •2. Critical clinical condition.
- •3. Congenital heart disease or cardiac arrhythmias.
研究者
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