Sporadic Amyotrophic Lateral Sclerosis Clinical Trial
— Fibro-ALSOfficial title:
Bioenergetics and Protein Metabolism in Sporadic Amyotrophic Lateral Sclerosis
Verified date | November 2016 |
Source | University Hospital, Angers |
Contact | n/a |
Is FDA regulated | No |
Health authority | France: Ministry of Health |
Study type | Interventional |
INTRODUCTION: Amyotrophic Lateral Sclerosis (ALS) is a degenerative disease of the motor
neurones of the brain and the spinal cord. The pathophysiological mechanisms of the disease
remain unknown. The average age of onset of ALS is about 60 years old, and the mean survival
of patients is about 2 years. The disease is responsible for a progressive paralysis leading
to death from respiratory failure. The only treatment available is the Riluzole, with a very
modest efficiency on the progression of the disease. ALS is the third neurodegenerative
disease, affecting 6000 persons in France, 150 000 in the world. Among the protagonists
involved in the occurrence of the disease, mitochondrial perturbations and protein
accumulations seem to be central elements.
OBJECTIVES: To precise the implication of energy and protein metabolism in the sporadic
forms of ALS, to identify potential biomarkers of the disease and to test new therapies.
METHODS: The investigators will study cell growth, bioenergetics, mitochondrial dynamics,
free-radicals production, presence of cytoplasmic inclusions, cytoskeleton structure and
stress response in primary skin fibroblasts obtained from sporadic ALS patients. The study
will be conducted over a period of three years in 3 centres specialized respectively in
motor neuron diseases, mitochondrial metabolism and neuronal cytoskeleton.
PROSPECTS: If the investigators achieve to identify differences between ALS fibroblasts and
controls, the results will be key elements to reinforce the hypothesis of a systemic disease
with an important metabolic participation, to better define ALS pathophysiological
mechanisms, to find potential biomarkers and to test new therapies.
Status | Not yet recruiting |
Enrollment | 30 |
Est. completion date | November 2019 |
Est. primary completion date | November 2017 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | Both |
Age group | 18 Years to 80 Years |
Eligibility |
Inclusion criteria : - a free signed an written consent - aged between 18 and 80 years old - ALS defined by El Escorial Criteria - normal neurological examination Exclusion criteria: - comorbidity or treatment susceptible to impact on metabolism - differential diagnosis suspected, early and late forms (<6 month > 3 years) - ALS not defined by El Escorial Criteria - withdrawal of consent to participate |
Allocation: Non-Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Basic Science
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
University Hospital, Angers |
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Kinetics of fibroblast growth | The growth rates of the fibroblast lines | 24 hours | No |
Secondary | Mitochondrial metabolism | the breath parameters of fibroblast will be precisely studied by oxygraphy, a method consisting in measuring the oxygen consumption rates of fibroblasts in different metabolic phases | 24 hours | No |
Secondary | Protéic metabolism | The presence of intracytoplasmic inclusion of target protein will be sought. | 24 hours | No |
Secondary | stress in senescence answer | the answer due to the stress of cell lines in hypoxia and nutritionnal lack will be studied. | 24 hours | No |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
NCT02781454 -
Mexiletine in Sporadic Amyotrophic Lateral Sclerosis
|
Phase 2 | |
Completed |
NCT01849770 -
Mexiletine in Sporadic Amyotrophic Lateral Sclerosis (SALS)
|
Phase 2 |