Therapeutic Metabolic Intervention in Patients With Spastic Paraplegia SPG5
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Change of 27-hydroxycholesterol blood level after 2 months of treatment for each treatment
研究概览
简要总结
The purpose of this project is to study the efficacy of three candidate molecules (Xenbilox, Tahor and Resveratrol) in order to decrease the production of oxysterols by reducing the synthesis of cholesterol and/or regulate the production of bile acids and/or enabling neuroprotective action within the motor neuron.
详细描述
The primary objective of the study is:
- decrease the accumulation of metabolites which can have a negative impact on neurological and systemic function of patients with SPG5.
The secondary objectives of the study are:
- confirm the clinical and biological tolerance of the different candidate molecules under study
- improve the serum bile acid profile of patients with SPG5
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 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
- •patients that have confirmed through genetic testing their status as carriers of 2 mutations in the CYP7B1 gene
- •age ≥ 18 years
- •patients that have signed the informed consent form
- •presence of health care coverage
排除标准
- •known hypersensitvity to chenodeoxycholic acid, atorvastatin, resveratrol or to any of their byproducts
- •cholesterol lowering medications other than the study treatment
- •hepatic failure with transaminases >3 times the normal level
- •progressive biliary pathology
- •chronic diarrhea
- •serious mental illness
- •significant comorbid neurological disorder
- •incapacity to understand information about the protocol
- •unwilling or unable to participate in any part of the study
- •participation in another clinical trial during the study period
- •person deprived of liberty by judicial or administrative decision
- •adult subject under legal protection or unable to consent
- •pregnant or breastfeeding women
- •lack of health care coverage
- •absence of a signed informed consent form
研究组 & 干预措施
Xenbilox
Xenbilox (chenodeoxycholic acid) 1000mg capsule by mouth every day for 2 months
干预措施: Xenbilox (Drug)
Resveratrol
Resveratrol 80mg capsule by mouth every day for 2 months
干预措施: Resveratrol (Dietary Supplement)
Tahor
Tahor (atorvastatin) 40mg tablet by mouth every day for 2 months
干预措施: Tahor (Drug)
结局指标
主要结局
Change of 27-hydroxycholesterol blood level after 2 months of treatment for each treatment
时间窗: 2 months
27-hydroxycholesterol will be measured on fasten blood samples for the 12 patients included before and after the 2-month treatment period for each treatment
次要结局
- Number of partcipants with adverse events related with standard biological parameters(18 months)
- Change of 25-hydroxycholesterol blood level after 2 months of treatment for each treatment(2 months)
- Number of participants with adverse events related with digestive problems(18 months)
- Modification of the serum acid profile after a 2-month treatment measured in blood and urine samples(2 months)