Study of Predictive Factors of Progression of Motor Neurone Disease

  • STATUS
    Recruiting
  • End date
    Jan 13, 2025
  • participants needed
    1000
  • sponsor
    University Hospital, Lille
Updated on 13 July 2020
Investigator
David Devos, MD,PhD
Primary Contact
H pital Dupuytren (0.0 mi away) Contact
+20 other location
neurological disorders
spasm
non-invasive ventilation
deficit
neurodegenerative disorders
neurological disorder
primary lateral sclerosis
motor neuron disease
fasciculation
amyotrophic lateral sclerosis

Summary

Amyotrophic lateral sclerosis (ALS) is a complex polymorph and devastating neurodegenerative disease. Although the pathophysiological mechanisms underlying the development of ALS remain to be fully elucidated, there have been significant advances in the understanding of ALS pathogenesis, with evidence emerging of a complex interaction between genetic factors and dysfunction of vital molecular pathways. However, the numerous randomized clinical trials (RCT) for ALS have failed to generate improved drug treatments. Biomarkers able to bring prognostic value and to distinguish the different endophenotypes of this polymorphic disease could help to better select clusters of patients in order to improve the RCT outcomes. However, little progress has been made in the development of viable diagnostic, prognostic and monitoring markers. This could be explained by common shortcomings, such as relatively small sample sizes, statistically underpowered study designs, lack of disease controls and poorly characterized patient cohorts. It is yet crucial that the investigators further develop and validate ALS biomarkers and incorporate these biomarkers into the drug development pipeline for ALS.

The aim of the present study is therefore to determine the clinical, biological, imaging, and electrophysiological biomarkers of prognosis of survival without events (i.e. severe comorbidity, 24 hours of non invasive ventilation, tracheotomy). This is a prospective observational multicentric French study of a cohort of 1000 ALS patients.

This large multimodal database will be open for international fruitful scientific collaborations.

Description

This is a prospective observational multicentric French study of a cohort of 1000 ALS patients, 100 neurological controls and 200 healthy controls followed from the first signs to the end of the disease.

The aim of the present study is therefore to determine the clinical, biological, imaging, and electrophysiological biomarkers of prognosis of survival without events (i.e. severe comorbidity, 24 hours of non invasive ventilation, tracheotomy).

Secondary objectives will notably include i) the biomarkers of disease progression ii) biomarkers of diagnosis as compared with controls iii) the determination of the different endophenotypes according to the prognosis, the genetic profiles and the initial clinical presentations. Criteria of assessment will notably include detailed medical history, habitus, past and present treatments, vascular risk factors, ALSFRS-R, muscular testing, respiratory parameters including early nocturnal saturation and apneal profile, the detailed and predetermined clinical presentations, extensive cognitive and behavioral examination, extensive blood, cerebrospinal fluid, urines biological tests, genetic analyses, multiple brain and spinal MRI sequences, and electrophysiological tests (i.e. electromyogram, MUNIX, triple stimulation). Invasive tests will be optional (i.e. lumbar puncture, skin biopsies, muscular biopsies).

The large number of patients will allow in depth statistical analyses, notably to establish decisional trees (CHAID).

Details
Treatment biomarkers (composite analysis)
Clinical Study IdentifierNCT02360891
SponsorUniversity Hospital, Lille
Last Modified on13 July 2020

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Eligibility

Yes No Not Sure

Inclusion Criteria

Is your age between 18 yrs and 85 yrs?
Gender: Male or Female
Do you have any of these conditions: Amyotrophic Lateral Sclerosis (ALS) or Amyotrophic Lateral Sclerosis or Myelopathy or Spinal Cord Disorders or Motor Neuron Disease?
Patients with suspicion of Amyotrophic lateral sclerosis
Since the first signs (cramps fasciculation) or first deficit at the diagnosis work up
Patient older than 18 years
Patient able to provide informed consent
Inclusion Criteria for healthy controls
Subjects older than 18 years (matched population for age and sex with ALS)
Neurological testing and examination showing no neurological disorders
Not having severe disease or life- functional prognosis
Inclusion Criteria for neurological controls
Patients having a typical neurodegenerative diseases other than ALS (Parkinson's disease, Alzheimer's disease)
Not having severe disease or life- functional prognosis
Patient older than 18 years (matched population for age and sex with ALS)
Patient able to provide informed consent

Exclusion Criteria

Subjects younger than 18 years
Patient unable to provide informed consent
Having severe disease or life- functional prognosis
Contraindications MRI
Clear my responses

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