New Therapeutic Strategy in ALS Based on Metabolic Status and Associated Metabolic Pathways.

NCT03984708 | Completed

• 2 other identifiers: DR1801352019-A00649-48
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 2

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."

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Amyotrophic Lateral Sclerosis; Metabolomics; Transcriptomics; Biomarkers; Fibroblast

Outcome Measures

Primary Outcomes (2)

• 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

  Baseline

• 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

  Baseline

Secondary Outcomes (1)

• Expression levels of targeted molecules using transcriptomics

  Baseline

Study Arms (2)

Case group

OTHER

The intervention, specific to the study, is to take samples at baseline on patients with Amyotrophic Lateral Sclerosis

Other: Samples; Other: Indirect calorimetry; Other: Electrical bioimpedance

Control group

OTHER

The intervention, specific to the study, is to take samples at baseline on patients without neurological disease

Other: Samples

Interventions

Electrical bioimpedance OTHER

Measurement of electrical bioimpedance

Case group

Samples OTHER

Blood sample, skin biopsy

Case group; Control group

Indirect calorimetry OTHER

Measurement of energy expenditure by indirect calorimetry

Case group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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:
• Age ≥ 18 years and ≥ 75 years
• No neuronal disease
• Patients affiliated to social security scheme
• Informed consent signed by the patient
• Pregnant or breastfeeding women

Sponsors & Collaborators

• University Hospital, Tours: lead

Study Sites (2)

Neurology Department, University Hospital, Limoges

Limoges, 87042, France

Location

Neurology Department, University Hospitla, Tours

Tours, 37044, France

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Sampling Studies; Calorimetry, Indirect

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Epidemiologic Study Characteristics; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health; Calorimetry; Chemistry Techniques, Analytical

Study Officials

• Hélène BLASCO, MD-PhD

  University Hospital, Tours

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: HEALTH SERVICES RESEARCH
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 4, 2019
• First Posted: June 13, 2019
• Study Start: January 27, 2020
• Primary Completion: January 10, 2023
• Study Completion: March 5, 2024
• Last Updated: December 30, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2)