ETUDE PREUVE DE CONCEPT D'UNE APPROCHE ANAPLEROTIQUE UTILISANT LA SPECTROSCOPIE RMN CEREBRALE AU PHOSPHORE-31 DANS LA MALADIE DE HUNTINGTON
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr
研究概览
简要总结
The purpose of this project is to study the efficacy of an anaplerotic treatment on brain energy profile evolution at an early stage of the Huntington disease.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •5 < UHDRS < 50
- •Age > 18 years
- •Ability to undergo MR scanning
- •Covered by french social security
排除标准
- •Evidence of psychiatric disorder
- •Attendant neurological disorder
- •Contraindications to MRI (claustrophobia, metallic or material implants)
- •Severe head injury
- •Unable to understand the protocol
- •Pregnancy
- •Failure to give informed consent
- •Subjects with exclusion criteria required by french law (e.g. subjects who require a legally authorized representative to obtain consent)
- •Unwillingness to be informed in case of abnormal MRI
研究组 & 干预措施
Triheptanoin 1g/kg/day
All patients received Triheptanoin oil at 1g/kg/day during 1 month
干预措施: Triheptanoin 1g/kg/day (Drug)
结局指标
主要结局
Ratio of Inorganic Phosphate (Pi) Over Phosphocreatine (PCr): Pi/PCr
时间窗: visit 1 (baseline), visit 2 (after 1 month of treatment)
The Pi/PCr Ratio is a measure of brain metabolism and it is an index of mitochondrial oxidative regulation. A 6-cm 31P transmit/receive surface coil (RAPID Biomedical GmbH, Rimpar, Germany) was used to collect free induction decays for 4 minutes at rest, 8 minutes during visual activation with 6-Hz red/black checkerboard flashes, and 8 minutes after stimulation. Subjects were able to focus on the flashes with a nonmagnetic mirror mounted above their eyes while all lights in the room were turned off. The Pi/PCr ratio was then calculated to determine brain response to cortical activation.
次要结局
- Correlation Between Primary Outcome Measure and Clinical Parameters(1 month)