Serum Neurofilaments in the Diagnosis of Amyotrophic Lateral Sclerosis
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorUniversity Hospital, Montpellier
About this trial
Amyotrophic lateral sclerosis (ALS) is a serious neurodegenerative disease, often difficult to diagnose due to symptoms similar to other neurological pathologies. Diagnosis can take up to 14 months, although the rapid progression of the disease requires early detection. At present, there is no validated biomarker to aid diagnosis. Serum neurofilaments light chain (NfL), markers of neuronal degeneration, show great potential to help diagnose ALS early and assess disease severity. Recent research has shown that measurement of NfL in the blood can differentiate ALS from other neurological disorders, and new technologies are increasingly making it possible to perform these tests clinically.
The study hypothesis is that NfL blood levels, measured using clinical analyzers, could improve early ALS diagnosis, optimize patient recruitment for therapeutic trials and accelerate the assessment of treatment efficacy.
The primary objective is to evaluate the sensitivity and specificity of serum NfL for the diagnosis and differential diagnosis of amyotrophic lateral sclerosis (ALS) in newly recruited patients referred to the ALS Reference Center at Montpellier University Hospital. The diagnosis is established according to the revised El Escorial diagnostic criteria (see Appendix). This diagnosis is determined independently of the serum NfL concentration.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Be at least 18 years of age
Be able to undergo blood sampling (however, blood sampling is part of the standard examination and will not be performed exclusively for this study).
Patients with suspected ALS
Disqualifiers
Pregnant or breast-feeding women
Patient deprived of liberty by judicial or administrative decision, or hospitalization under duress
Adult protected by law (guardianship, curatorship)
Patient unable to understand and read information and consent forms in French
Trial population
The study population consists of all patients referred to the rare disease reference center for amyotrophic lateral sclerosis (ALS) at CHU Montpellier for suspected ALS, regardless of the final diagnosis. These patients will be included prospectively as part of the diagnostic evaluation. The inclusion will be based on clinical suspicion of ALS, and the final diagnosis will be established according to the revised El Escorial criteria, independent of the NfL serum levels. This population will be monitored and analyzed for diagnostic performance and prognostic value of serum NfL levels.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Serum Neurofilament Serum NfL Measurement
Diagnostic testThe procedure involves taking an additional 6 ml blood sample (dry tube) during the first visit, in addition to the routine sample taken for diagnostic investigations. Serum levels of neurofilament light chain (NfL), a biomarker of neuronal damage, will be measured using ultrasensitive techniques (SIMOA, Lumipulse, Cobas). The aim is to assess the diagnostic performance of NfL levels in differentiating ALS from other neurodegenerative diseases, as well as their prognostic value in terms of survival and disease progression.
Treatment groups
138 Participants
are divided into 1 treatment groupGroup A: Patients Suspected of ALS1 intervention
Trial outcomes
Primary outcomes
1
Evaluate the diagnostic performance of blood NfL levels for the diagnosis of ALS
Evaluate the diagnostic performance of blood NfL levels for the diagnosis of ALS. Evaluation of the diagnostic performance of NfL blood levels (pg/mL) on samples taken during the patient inclusion visit. The final diagnosis will be established independently of the serum NfL levels at inclusion. The ALS diagnosis will be made according to the revised El Escorial criteria, which distinguish between definite, probable, clinically probable with paraclinical support, or possible ALS diagnoses.
Time frame
From baseline (Visit 0) up to 12 monthsSecondary outcomes
1
Functional decline
ALS Functional Rating Scale - Revised (ALSFRS-r) is administered regularly to evaluate the patient's functional decline, which will be correlated with serum NfL levels to assess the prognostic value of NfL in predicting disease progression.
Time frame
From enrollment to the end of follow-up, at least every 3 months during routine clinical care.2
Respiratory function
Pulmonary Function Test. FVC and SVC are important indicators of respiratory function, which typically declines as ALS progresses. These values will be correlated with NfL levels to investigate how early changes in NfL relate to respiratory decline.
Time frame
From enrollment to the end of follow-up, at least every 3 months during routine clinical care.3
Initiation of non-invasive ventilation
The need for NIV will be monitored and correlated with serum NfL levels to determine whether elevated NfL levels correlate with an earlier need for NIV, suggesting a more rapid disease progression.
Time frame
From enrollment to the end of follow-up, at least every 3 months during routine clinical care.4
Overall survival
This endpoint will examine the correlation between serum NfL levels and patient survival. The hypothesis is that higher levels of NfL could correlate with shorter survival times due to more rapid neurodegeneration.
Time frame
From enrollment to the end of follow-up, at least every 3 months during routine clinical care.Locations
Sponsors and contacts
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Source ClinicalTrials.gov
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