NCT02014883已完成2 期
Phase II Open Label Study Using Triheptanoin in Patients With Glucose Type 1 Transporter Deficiency GLUT1-DS
Institut National de la Santé Et de la Recherche Médicale, France1 个研究点 分布在 1 个国家目标入组 20 人开始时间: 2013年12月4日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Number of paroxystic events
研究概览
简要总结
The purpose of this project is to study the efficacy of triheptanoin oil in patients with GLUT1 deficiency syndrome.
详细描述
The primary objective of the study is:
- to evaluate the capacity of triheptanoïn to improve the condition of patients with GLUT1-DS
The secondary objectives of the study are:
- to confirm the short-term safety of triheptanoïn therapy in patients with GLUT1-DS
- to evaluate the short-term effects of triheptanoïn treatment on motor function, autonomy, quality of life and clinical signs of patients with GLUT1-DS
- to evaluate the effect of triheptanoïn on brain energy metabolism using non-invasive 31P-MRS spectroscopy after activation of the occipital cortex in order to measure the levels of high-energy phosphates (such as ATP and phosphocreatine)
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Mutation in SLC2A1 gene
- •Age > 3 years
- •Patient with history/frequency of seizures or movement disorders documented at least 3 months prior to the beginning of the study
- •Covered by french social security
- •Patients who freely agree to participate in this study and understand the nature, risks and benefits of this study and give their written informed consent. (In addition to the requirement for the consent of parents or the legal representative, adolescents can provide additional informed consent to participate in clinical trials)
排除标准
- •Evidence of psychiatric disorder
- •Attendant neurological disorder
- •Comorbid medical condition that would render them unsuitable for the study, e.g. HIV, diabetes
- •Pregnant or parturient or lactating women
- •Unwillingness to be informed in case of abnormal MRI
- •Failure to give written informed consent
- •Unable to understand the protocol
- •Unable to participate to the whole study
- •Absence of signed informed consent
- •Persons deprived of their liberty by judicial or administrative decision
- •Person subject to an exclusion period for another research
- •Subjects with exclusion criteria required by french law
研究组 & 干预措施
GLUT1 DS
Experimental
干预措施: GLUT1 DS (Drug)
结局指标
主要结局
Number of paroxystic events
时间窗: 6 months
The number of paroxystic events, in particular abnormal movements, will be collected during trihepatnoin treatment.
次要结局
- 6 minutes walk test(6 months)
- Brain 31phosphorus magnetic resonance spectroscopy(6 months)
- 9 hole Peg board(6 months)
- Clinical Global Impression Scales(6 months)
- Schwab-England scale(6 months)
- Vineland Scale(6 months)
- Fatigue Severity Scale(6 months)
- Fatigue Visual Scale(6 months)
- Safety(6 months)
研究者
研究点 (1)
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