New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways.
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 9
- 试验地点
- 4
- 主要终点
- Metabolic signature of blood : concentrations of molecules detected by mass spectrometry
研究概览
简要总结
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that affects central and peripheral motor neurons. None of the clinical trials conducted have been clearly successful and the disease remains incurable, putting patients' vital prognosis at risk in the medium term. An alteration of the basal metabolism leading to hypermetabolism has been described in several articles in the literature. The causes of this hypermetabolism and the precise exploration of the metabolic pathways involved are still poorly understood. The fibroblasts of ALS patients may be the site of some metabolic disturbances in this disease with a hypothetical specific basal metabolic profile. These cells are adapted to different metabolic explorations such as omnic approaches. Superficial skin biopsy followed by fibroblast culture can provide a considerable biobank. This cellular richness will allow us, in ALS patients and their controls, to perform metabolomic and lipidomic approaches, as well as the quantification transcriptomic approach."
详细描述
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that affects central and peripheral motor neurons. None of the clinical trials conducted have been clearly successful and the disease remains incurable, putting patients' vital prognosis at risk in the medium term. An alteration of the basal metabolism leading to hypermetabolism has been described in several articles in the literature. The causes of this hypermetabolism and the precise exploration of the metabolic pathways involved are still poorly understood. The fibroblasts of ALS patients may be the site of some metabolic disturbances in this disease with a hypothetical specific basal metabolic profile. These cells are adapted to different metabolic explorations such as omnic approaches. Superficial skin biopsy followed by fibroblast culture can provide a considerable biobank. This cellular richness will allow us, in ALS patients and their controls, to perform experiments for the quantification of metabolites by metabolic and lipidomic approaches, as well as the quantification of mRNAs and the rate of gene transcription by a transcriptomic approach.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Health Services Research
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 75 Years(Adult, Older Adult)
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •Age ≥ 18 years and ≥ 75 years
- •ALS according to the El Escorial criteria
- •Diagnosis of ALS < 6 months
- •Symptoms onset < 2 years
- •Patients affiliated to social security scheme
- •Informed consent signed by the patient
排除标准
- •Pregnant or breastfeeding women
- •Contraindication to biopsy
- •Contraindication to local anesthesia
- •Treatment with oral or injectable anticoagulants, antiplatelet (except aspirin)
- •Unbalanced Diabetes
- •Systemic corticosteroid treatment
- •Dermatological diseases of the fibroblast
- •Skin cancer
- •Protection measure for guardianship or curatorship
- •Control group selection criteria:
- •Inclusion Criteria:
- •Age ≥ 18 years and ≥ 75 years
- •No neuronal disease
- •Patients affiliated to social security scheme
- •Informed consent signed by the patient
- •Exclusion Criteria:
- •Pregnant or breastfeeding women
- •Contraindication to biopsy
- •Treatment with oral or injectable anticoagulants, antiplatelet (except aspirin)
- •Unbalanced Diabetes
- •Systemic corticosteroid treatment
- •Dermatological diseases of the fibroblast
- •Skin cancer
- •Protection measure for guardianship or curatorship
结局指标
主要结局
Metabolic signature of blood : concentrations of molecules detected by mass spectrometry
时间窗: Baseline
The metabolomic profile of blood represents the combination of the different molecules detected/quantified by mass spectrometry
Metabolic signature of fibroblast : concentrations of molecules detected by mass spectrometry
时间窗: Baseline
The metabolomic profile of fibroblast represents the combination of the different molecules detected/quantified by mass spectrometry
次要结局
- Expression levels of targeted molecules using transcriptomics(Baseline)
