Amyotrophic Lateral SclerosisNfL-ALSNCT06201650
Neurofilament Light Chain in Amyotrophic Lateral Sclerosis
An observational study of Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Charite University, Berlin, Germany at 19 sites in 2 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- What is being tested?
- Neurofilament light chain
- How many people
- 3,000 participants
- Where
- 19 sitesin 2 countries
- Study dates
- 11 Nov 2020to 31 Dec 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: patients with clinical diagnosis of amyotrophic lateral sclerosis
Who may be able to join
- 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
Who cannot join
- Unwillingness to store and share pseudonymized medical data collected in the study
- Evaluation by the investigator, which excludes participation
Trial locations and contacts
19 sites in 2 countries.
19 sites in 2 countries
Germany18 sites, 18 recruiting
- Recruiting
Vivantes Klinikum Kaulsdorf
Berlin, 12621
Christoph Richter, Dr
Christoph Richter, Dr
- Recruiting
Charité Universitätsmedizin Berlin
Berlin, 13353
Thomas Meyer, Prof Dr, thomas.meyer@charite.de
Erma Salkic, erma.salkic@charite.de
- Recruiting
Berufsgenossenschaftliches Universitätsklinikum Bergmannsheil gGmbH
Bochum, 44789
Ute Weyen, Dr
Ute Weyen, Dr
- Recruiting
Universitätsklinikum Bonn
Bonn, 53127
Patrick Weydt, PD Dr
Patrick Weydt, PD Dr
- Recruiting
Technische Universität Dresden
Dresden, 01062
René Günther, Dr
René Günther, Dr
- Recruiting
Alfried Krupp von Bohlen und Halbach Krankenhaus gGmbH
Essen, 45131
Torsten Grehl, Dr
Torsten Grehl, Dr
- Recruiting
Universitätsklinikum Essen
Essen, 45147
Tim Hagenacker, Prof Dr
Tim Hagenacker, Prof Dr
- Recruiting
Georg-August-Universität Göttingen Universitätsmedizin Göttingen
Göttingen, 37075
Jan Koch, PD Dr
Jan Koch, PD Dr
- Recruiting
Medizinische Hochschule Hannover
Hanover, 30625
Susanne Petri, Prof Dr
Susanne Petri, Prof Dr
- Recruiting
Universitätsklinikum Jena
Jena, 07743
Robert Steinbach, Dr
- Recruiting
Universität Leipzig
Leipzig, 04103
Petra Baum, PD Dr
Petra Baum, PD Dr
- Recruiting
Universitätsklinikum Schleswig-Holstein
Lübeck, 23538
Julian Grosskreutz, Prof Dr
Julian Grosskreutz, Prof Dr
- Recruiting
Universität Heidelberg Medizinische Fakultät Mannheim
Mannheim, 68167
Jochen Weishaupt, Prof Dr
Jochen Weishaupt, Prof Dr
- Recruiting
Klinikum rechts der Isar der Technischen Universität München
München, 81675
Paul Lingor, Prof Dr
Paul Lingor, Prof Dr
- Recruiting
Westfälische Wilhelms-Universität Münster
Münster, 48149
Matthias Boentert, PD DR
Matthias Boentert, PD Dr
- Recruiting
Universitätsklinikum Regensburg
Regensburg, 93053
Zacharias Kohl, PD Dr
Zacharias Kohl, PD Dr
- Recruiting
Universitätsmedizin Rostock
Rostock, 18057
Andreas Hermann, Prof. Dr. med. Dr. rer. med.
Andreas Hermann, Prof Dr Dr
- Recruiting
Universitätsklinikum Ulm
Ulm, 89081
Johannes Dorst, Prof Dr
Johannes Dorst, Prof Dr
Austria1 site, 1 recruiting
- Not yet recruiting
Medizinische Universität Innsbruck
Innsbruck, 6020
Wolfgang Löscher, Prof Dr
Wolfgang Löscher, Prof Dr
About this study
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
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.
Read the 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
- Being tested
- Neurofilament light chain
- Keywords
- ALS, Amyotrophic lateral sclerosis, Neurofilament light chain, NfL, ALS Funktional Rating Scale, Progression, biomarker, treatment marker, therapeutic marker
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Charcot-Marie-Tooth disease, Type 1F, Disease Progression
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Neurofilament light chainDiagnostic test
Neurofilament light chain as biomarker for amyotrophic lateral sclerosis
What is being measured
The primary outcome is the main question the study is designed to answer.
correlation of serum neurofilament light chain to ALS progression
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)
Measured: 2020-2024
correlation of serum neurofilament light chain with ALS phenotypes
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
Measured: 2020-2024
correlation of serum neurofilament light chain with ALS treatment options
correlation of serum neurofilament light chain to ALS interventions such as treatment with tofersen and other medicines
Measured: 2020-2024
Secondary outcomes (1)
correlation of serum neurofilament light chain with non-pharmacologic ALS interventions
correlation of serum neurofilament light chain with non-pharmacologic ALS interventions including nutrition or ventilation treatment
Measured: 2022-2024
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Jan 2024
- Study start11 Nov 2020
- Primary completion31 Dec 2024 (estimated)
- Study completion31 Dec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Charite University, Berlin, GermanyThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 3,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Charite University, Berlin, Germany
- Sponsor type
- University, hospital or other
- Collaborators
- Boris Canessa ALS Stiftung
- Responsible party
- Principal investigator, Thomas Meyer, MD, Charite University, Berlin, Germany
- Investigators
- Thomas Meyer, MD, Principal investigator, Charite University, Berlin, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06201650
- Sponsor's protocol ID
- NfL-ALS
- Acronym
- NfL-ALS
- Status last verified
- Dec 2023
- First submitted
- 1 Apr 2023
- Last updated
- 11 Jan 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 11 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06201650
- Acronym / sponsor ID
- NfL-ALS / NfL-ALS
- Keywords
- ALS, Amyotrophic lateral sclerosis, Neurofilament light chain, NfL, ALS Funktional Rating Scale, Progression, biomarker, treatment marker
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 3,000 participants (estimated)
- Lead sponsor
- Charite University, Berlin, GermanyUniversity, hospital or other
- Collaborators
- Boris Canessa ALS Stiftung
- Study start
- 11 Nov 2020 (actual)
- Primary completion
- 31 Dec 2024 (estimated)
- Study completion
- 31 Dec 2024 (estimated)
- First posted
- 11 Jan 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- patients with clinical diagnosis of amyotrophic lateral sclerosis
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (2 listed)
- Unwillingness to store and share pseudonymized medical data collected in the study
- Evaluation by the investigator, which excludes participation
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Neurofilament light chainNeurofilament light chain as biomarker for amyotrophic lateral sclerosis | Diagnostic test | – |
Primary outcome
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)
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
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
1 secondary outcome
correlation of serum neurofilament light chain with non-pharmacologic ALS interventions
Time frame: 2022-2024
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jan 2024.
- Website of principle investigatorLink provided by the sponsor.
- Website of funding organizationLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Thomas Meyer, MD principal investigator
Charite University, Berlin, Germany
19 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by Charite University, Berlin, Germany at 19 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 11 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in Germany, Austria. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Thomas Meyer, MD (004930450560028, thomas.meyer@charite.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
correlation of serum neurofilament light chain to ALS progression (time frame: 2020-2024). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jan 2024.