New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways. (METABOCALS)
Primary Purpose
Amyotrophic Lateral Sclerosis
Status
Recruiting
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Samples
Indirect calorimetry
Electrical bioimpedance
Sponsored by
About this trial
This is an interventional health services research trial for Amyotrophic Lateral Sclerosis focused on measuring Amyotrophic Lateral Sclerosis, Metabolomics, Transcriptomics, Biomarkers, Fibroblast
Eligibility 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
Sites / Locations
- Neurology Department, University Hospital, LimogesRecruiting
- Neurology Department, University Hospitla, ToursRecruiting
Arms of the Study
Arm 1
Arm 2
Arm Type
Other
Other
Arm Label
Case group
Control group
Arm Description
The intervention, specific to the study, is to take samples at baseline on patients with Amyotrophic Lateral Sclerosis
The intervention, specific to the study, is to take samples at baseline on patients without neurological disease
Outcomes
Primary Outcome Measures
Metabolic signature of fibroblast : concentrations of molecules detected by mass spectrometry
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
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
Choice of molecules based on results obtained by metabolomics approaches
Full Information
NCT ID
NCT03984708
First Posted
June 4, 2019
Last Updated
September 29, 2021
Sponsor
University Hospital, Tours
1. Study Identification
Unique Protocol Identification Number
NCT03984708
Brief Title
New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways.
Acronym
METABOCALS
Official Title
New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways.
Study Type
Interventional
2. Study Status
Record Verification Date
September 2021
Overall Recruitment Status
Recruiting
Study Start Date
January 27, 2020 (Actual)
Primary Completion Date
January 2023 (Anticipated)
Study Completion Date
January 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University Hospital, Tours
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. 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."
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
Amyotrophic Lateral Sclerosis, Metabolomics, Transcriptomics, Biomarkers, Fibroblast
7. Study Design
Primary Purpose
Health Services Research
Study Phase
Not Applicable
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
90 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Case group
Arm Type
Other
Arm Description
The intervention, specific to the study, is to take samples at baseline on patients with Amyotrophic Lateral Sclerosis
Arm Title
Control group
Arm Type
Other
Arm Description
The intervention, specific to the study, is to take samples at baseline on patients without neurological disease
Intervention Type
Other
Intervention Name(s)
Samples
Intervention Description
Blood sample, skin biopsy
Intervention Type
Other
Intervention Name(s)
Indirect calorimetry
Intervention Description
Measurement of energy expenditure by indirect calorimetry
Intervention Type
Other
Intervention Name(s)
Electrical bioimpedance
Intervention Description
Measurement of electrical bioimpedance
Primary Outcome Measure Information:
Title
Metabolic signature of fibroblast : concentrations of molecules detected by mass spectrometry
Description
The metabolomic profile of fibroblast represents the combination of the different molecules detected/quantified by mass spectrometry
Time Frame
Baseline
Title
Metabolic signature of blood : concentrations of molecules detected by mass spectrometry
Description
The metabolomic profile of blood represents the combination of the different molecules detected/quantified by mass spectrometry
Time Frame
Baseline
Secondary Outcome Measure Information:
Title
Expression levels of targeted molecules using transcriptomics
Description
Choice of molecules based on results obtained by metabolomics approaches
Time Frame
Baseline
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Hélène BLASCO, MD-PhD
Phone
02.34.37.89.11
Ext
+33
Email
helene.blasco@univ-tours.fr
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hélène BLASCO, MD-PhD
Organizational Affiliation
University Hospital, Tours
Official's Role
Study Director
Facility Information:
Facility Name
Neurology Department, University Hospital, Limoges
City
Limoges
ZIP/Postal Code
87042
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Philippe COURATIER, MD-PhD
First Name & Middle Initial & Last Name & Degree
Philippe COURATIER, MD-PhD
Facility Name
Neurology Department, University Hospitla, Tours
City
Tours
ZIP/Postal Code
37044
Country
France
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Philippe CORCIA, MD-PhD
First Name & Middle Initial & Last Name & Degree
Philippe CORCIA, MD-PhD
12. IPD Sharing Statement
Plan to Share IPD
No
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New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways.
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