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出境医 / 临床实验 / New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways. (METABOCALS)
New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways. (METABOCALS)
Study Description
Brief Summary:
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."
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Amyotrophic Lateral Sclerosis | Other: Samples Other: Indirect calorimetry Other: Electrical bioimpedance | Not Applicable |
Detailed Description:
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.
Study Design
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 90 participants |
| Allocation: | Non-Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Health Services Research |
| Official Title: | New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways. |
| Actual Study Start Date : | January 27, 2020 |
| Estimated Primary Completion Date : | June 2021 |
| Estimated Study Completion Date : | June 2022 |
Arms and Interventions
| Arm | Intervention/treatment |
|---|---|
|
Case group
The intervention, specific to the study, is to take samples at baseline on patients with Amyotrophic Lateral Sclerosis
|
Other: Samples
Blood sample, skin biopsy
Other: Indirect calorimetry Measurement of energy expenditure by indirect calorimetry
Other: Electrical bioimpedance Measurement of electrical bioimpedance
|
|
Control group
The intervention, specific to the study, is to take samples at baseline on patients without neurological disease
|
Other: Samples
Blood sample, skin biopsy
|
Outcome Measures
Primary Outcome Measures :
- Metabolic signature of fibroblast : concentrations of molecules detected by mass spectrometry [ Time Frame: Baseline ]The metabolomic profile of fibroblast represents the combination of the different molecules detected/quantified by mass spectrometry
- Metabolic signature of blood : concentrations of molecules detected by mass spectrometry [ Time Frame: Baseline ]The metabolomic profile of blood represents the combination of the different molecules detected/quantified by mass spectrometry
Secondary Outcome Measures :
- Expression levels of targeted molecules using transcriptomics [ Time Frame: Baseline ]Choice of molecules based on results obtained by metabolomics approaches
Eligibility Criteria
| Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | Yes |
Criteria
Case group selection criteria:
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Contacts and Locations
Contacts
| Contact: Hélène BLASCO, MD-PhD | 02.34.37.89.11 ext +33 | helene.blasco@univ-tours.fr |
Locations
| France | |
| Neurology Department, University Hospital, Limoges | Recruiting |
| Limoges, France, 87042 | |
| Contact: Philippe COURATIER, MD-PhD | |
| Principal Investigator: Philippe COURATIER, MD-PhD | |
| Neurology Department, University Hospitla, Tours | Recruiting |
| Tours, France, 37044 | |
| Contact: Philippe CORCIA, MD-PhD | |
| Principal Investigator: Philippe CORCIA, MD-PhD | |
Sponsors and Collaborators
University Hospital, Tours
Investigators
| Study Director: | Hélène BLASCO, MD-PhD | University Hospital, Tours |
| Tracking Information | |||||
|---|---|---|---|---|---|
| First Submitted Date ICMJE | June 4, 2019 | ||||
| First Posted Date ICMJE | June 13, 2019 | ||||
| Last Update Posted Date | April 28, 2021 | ||||
| Actual Study Start Date ICMJE | January 27, 2020 | ||||
| Estimated Primary Completion Date | June 2021 (Final data collection date for primary outcome measure) | ||||
| Current Primary Outcome Measures ICMJE |
|
||||
| Original Primary Outcome Measures ICMJE | Same as current | ||||
| Change History | |||||
| Current Secondary Outcome Measures ICMJE |
Expression levels of targeted molecules using transcriptomics [ Time Frame: Baseline ] Choice of molecules based on results obtained by metabolomics approaches
|
||||
| Original Secondary Outcome Measures ICMJE | Same as current | ||||
| Current Other Pre-specified Outcome Measures | Not Provided | ||||
| Original Other Pre-specified Outcome Measures | Not Provided | ||||
| Descriptive Information | |||||
| Brief Title ICMJE | New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways. | ||||
| Official Title ICMJE | New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways. | ||||
| Brief Summary | 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." | ||||
| Detailed Description | 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. | ||||
| Study Type ICMJE | Interventional | ||||
| Study Phase ICMJE | Not Applicable | ||||
| Study Design ICMJE | Allocation: Non-Randomized Intervention Model: Parallel Assignment Masking: None (Open Label) Primary Purpose: Health Services Research |
||||
| Condition ICMJE | Amyotrophic Lateral Sclerosis | ||||
| Intervention ICMJE |
|
||||
| Study Arms ICMJE |
|
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| Publications * | Not Provided | ||||
|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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| Recruitment Information | |||||
| Recruitment Status ICMJE | Recruiting | ||||
| Estimated Enrollment ICMJE |
90 | ||||
| Original Estimated Enrollment ICMJE | Same as current | ||||
| Estimated Study Completion Date ICMJE | June 2022 | ||||
| Estimated Primary Completion Date | June 2021 (Final data collection date for primary outcome measure) | ||||
| Eligibility Criteria ICMJE |
Case group selection criteria: Inclusion Criteria:
Exclusion Criteria:
Control group selection criteria: Inclusion Criteria:
Exclusion Criteria:
|
||||
| Sex/Gender ICMJE |
|
||||
| Ages ICMJE | 18 Years to 75 Years (Adult, Older Adult) | ||||
| Accepts Healthy Volunteers ICMJE | Yes | ||||
| Contacts ICMJE |
|
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| Listed Location Countries ICMJE | France | ||||
| Removed Location Countries | |||||
| Administrative Information | |||||
| NCT Number ICMJE | NCT03984708 | ||||
| Other Study ID Numbers ICMJE | DR180135 2019-A00649-48 ( Other Identifier: IdRCB ) |
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| Has Data Monitoring Committee | No | ||||
| U.S. FDA-regulated Product |
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| IPD Sharing Statement ICMJE |
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| Responsible Party | University Hospital, Tours | ||||
| Study Sponsor ICMJE | University Hospital, Tours | ||||
| Collaborators ICMJE | Not Provided | ||||
| Investigators ICMJE |
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| PRS Account | University Hospital, Tours | ||||
| Verification Date | April 2021 | ||||
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ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |
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