Study of Predictive Factors of Progression of Motor Neurone Disease Prognosis and Endophenotype Biomarkers French Database Set up

Study Purpose:

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.

Study Status:

Recruiting

Disease:

Amyotrophic Lateral Sclerosis , Motor Neuron Disease

Study Type:

Observational [Patient Registry]

Type of Intervention:

N/A

Intervention Name:

N/A

Placebo:

N/A

Phase:

N/A

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

David Devos, MD, PhD, University Hospital, Lille

Clinicaltrials.gov ID:

NCT02360891

Neals Affiliated?

No

Coordinating Center Contact Information

Study Sponsor:

University Hospital, Lille

Estimated Enrollment:

1000

Estimated Study Start Date:

01 / 12 / 2015

Estimated Study Completion Date:

01 / 01 / 2026

Posting Last Modified Date:

11 / 07 / 2022

Date Study Added to neals.org:

02 / 11 / 2015