This is the classic website, which will be retired eventually. Please visit the modernized ClinicalTrials.gov instead.
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Neurofilament Light Chain in Amyotrophic Lateral Sclerosis (NfL-ALS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT06201650
Recruitment Status : Recruiting
First Posted : January 11, 2024
Last Update Posted : January 11, 2024
Sponsor:
Collaborator:
Boris Canessa ALS Stiftung
Information provided by (Responsible Party):
Thomas Meyer, MD, Charite University, Berlin, Germany

Study Description
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
This study assesses the performance of serum neurofilament light chain (sNfL) in amyotrophic lateral sclerosis (ALS) in a wide range of disease courses, in terms of ALS progression, disease duration, and tracheostomy invasive ventilation (TIV). The aim of the research project is to investigate the correlation between NfL serum concentration and the natural course of the disease, the ALS progression rate, and specific phenotypes of ALS. Furthermore, the performance of NfL as a therapeutic biomarker will be studied. A systematic analysis of the NfL serum concentration in a cohort of 3,000 ALS patients using the Single Molecule Analysis method (SIMOA) will be performed. This analysis is carried out as a multi-center study.

Condition or disease Intervention/treatment
Amyotrophic Lateral Sclerosis Motor Neuron Disease Diagnostic Test: Neurofilament light chain

Detailed Description:

The aim of this study is to investigate the correlation between the NfL serum concentration and the natural course of the disease, the ALS progression rate as measured by the ALS functional rating scale (ALSFRS-R), and specific phenotypes of ALS. The results of the study will contribute to the assessment of disease progression and the prognosis making of ALS. Furthermore, the performance of NfL as a therapeutic marker of ALS medicines and non-pharmacologic treatment options will be investigated. A systematic analysis of the NfL serum concentration in an extended cohort of ALS patients using the Single Molecule Analysis method (SIMOA) will be performed.

Research objectives comprise:

  • Correlation of NfL with disease progression, including duration of ALS disease
  • Correlation of NfL with the course of ALS (classic ALS or variants in the motor neuron involvement or the regional propagation patterns)
  • Correlation of NfL with the progression rate of ALS

Cohorts on phenotypic variants:

The clinical phenotype of ALS will be differentiated according to the motor neuron involvement or regional propagation patterns of disease onset and clinical course.

ALS variants in relation to motor neuron involvement:

  • Typical ALS: clinical features for an affection of the 1st and 2nd motor neurons are present
  • Progressive muscular atrophy (PMA): only clinical features for an affection of the 2nd motor neuron are present
  • Spastic ALS: predominantly clinical features for an affection of the 1st motor neuron and fewer signs of an affection of the 2nd motor neuron
  • Primary lateral sclerosis (PLS): only clinical features for an affection of the 1st motor neuron are present

ALS variants in regional propagation patterns:

  • Typical form: paresis of the upper or lower extremities or the bulbar region as well as the spread of the paresis to other regions
  • Flail arm syndrome: primary and dominant paresis of the upper extremities and little or delayed spread of the paresis to other regions
  • Flail leg syndrome: primary and dominant paresis of the lower extremities and little or delayed spread of the paresis to other regions
  • Axial ALS: primary and dominant paresis of the trunk muscles and minor or delayed spread of the paresis to other regions
  • Progressive bulbar paralysis: primary and dominant paresis in the bulbar region and slight or delayed spread of the paresis to other regions
Study Design
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Layout table for study information
Study Type : Observational
Estimated Enrollment : 3000 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Performance of Serum Neurofilament Light Chain in a Wide Spectrum of Clinical Courses of Amyotrophic Lateral Sclerosis - a Cross-sectional and Longitudinal Multicenter Study
Actual Study Start Date : November 11, 2020
Estimated Primary Completion Date : December 31, 2024
Estimated Study Completion Date : December 31, 2024

Resource links provided by the National Library of Medicine


Groups and Cohorts
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Intervention Details:
  • Diagnostic Test: Neurofilament light chain
    Neurofilament light chain as biomarker for amyotrophic lateral sclerosis

Outcome Measures
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Primary Outcome Measures :
  1. correlation of serum neurofilament light chain to ALS progression [ Time Frame: 2020-2024 ]
    correlation of serum neurofilament light chain (NfL) with the ALS progression rate as measured by the revised form of the ALS function rating scale (ALSFRS-R)

  2. correlation of serum neurofilament light chain with ALS phenotypes [ Time Frame: 2020-2024 ]
    correlation of serum neurofilament light chain with ALS phenotypes in terms of type of onset and clinical variants including progressive muscle atrophy, primary lateral sclerosis, flail-arm syndrome, flail-leg syndrome and other phenotypes

  3. correlation of serum neurofilament light chain with ALS treatment options [ Time Frame: 2020-2024 ]
    correlation of serum neurofilament light chain to ALS interventions such as treatment with tofersen and other medicines


Secondary Outcome Measures :
  1. correlation of serum neurofilament light chain with non-pharmacologic ALS interventions [ Time Frame: 2022-2024 ]
    correlation of serum neurofilament light chain with non-pharmacologic ALS interventions including nutrition or ventilation treatment


Biospecimen Retention:   Samples Without DNA
Serum

Eligibility Criteria
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
patients with clinical diagnosis of amyotrophic lateral sclerosis
Criteria

Inclusion Criteria:

  • Diagnosis of amyotrophic lateral sclerosis including specific forms
  • Patient's informed consent to participate in this study
  • Minimum age of 18 years
  • Willingness for blood collection

Exclusion Criteria:

  • Unwillingness to store and share pseudonymized medical data collected in the study
  • Evaluation by the investigator, which excludes participation
Contacts and Locations
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT06201650


Contacts
Layout table for location contacts
Contact: Thomas Meyer, MD 004930450560028 thomas.meyer@charite.de
Contact: Péter Körtvélyessy, MD 004930450560028 peter.koertvelyessy@charite.de

Locations
Show Show 19 study locations
Sponsors and Collaborators
Charite University, Berlin, Germany
Boris Canessa ALS Stiftung
Investigators
Layout table for investigator information
Principal Investigator: Thomas Meyer, MD Charite University, Berlin, Germany
More Information
Go to 
Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
Additional Information:

Layout table for additonal information
Responsible Party: Thomas Meyer, MD, Prof. Dr. Thomas Meyer, Charite University, Berlin, Germany
ClinicalTrials.gov Identifier: NCT06201650    
Other Study ID Numbers: NfL-ALS
First Posted: January 11, 2024    Key Record Dates
Last Update Posted: January 11, 2024
Last Verified: December 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Thomas Meyer, MD, Charite University, Berlin, Germany:
ALS
Amyotrophic lateral sclerosis
Neurofilament light chain
NfL
ALS Funktional Rating Scale
 Progression
biomarker
treatment marker
therapeutic marker
Additional relevant MeSH terms:
Layout table for MeSH terms
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
 Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases