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

Study Purpose:

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."

Study Status:

Recruiting

Disease:

Amyotrophic Lateral Sclerosis

Study Type:

Interventional

Type of Intervention:

N/A

Intervention Name:

Samples, Indirect calorimetry, Electrical bioimpedance

Placebo:

Phase:

N/A

Study Chair(s)/Principal Investigator(s):

Hélène BLASCO, MD-PhD, University Hospital, Tours

Clinicaltrials.gov ID:

NCT03984708

Neals Affiliated?

No

Coordinating Center Contact Information

Hélène BLASCO, MD-PhD / email hidden; JavaScript is required / 02.34.37.89.11

Full Study 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 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 Sponsor:

University Hospital, Tours

Estimated Enrollment:

90

Estimated Study Start Date:

01 / 27 / 2020

Estimated Study Completion Date:

01 / 01 / 2024

Posting Last Modified Date:

09 / 30 / 2021

Date Study Added to neals.org:

06 / 13 / 2019

Minimum Age:

18 Years

Maximum Age:

75 Years

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

Neurology Department, University Hospital, Limoges | Recruiting

Philippe COURATIER, MD-PhD

Principal Investigator : Philippe COURATIER, MD-PhD

Limoges 87042
France

Neurology Department, University Hospitla, Tours | Recruiting

Philippe CORCIA, MD-PhD

Principal Investigator : Philippe CORCIA, MD-PhD

Tours 37044
France