Amyotrophic Lateral SclerosisPULSENCT02360891
Study of Predictive Factors of Progression of Motor Neurone Disease
An observational study of Amyotrophic Lateral Sclerosis and Motor Neuron Disease, run by University Hospital, Lille at 13 sites in France.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 85 yearsHealthy volunteers can take part
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
- What is being tested?
- biomarkers (composite analysis)
- How many people
- 500 participants
- Where
- 13 sitesFrance
- Study dates
- 12 Jan 2015to 30 Jun 2025
- How long will I take part?
- 10 Years
- 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 to 85 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: Multicentric French cohort of 1000 ALS patients, 100 neurological controls and 200 healthy controls
Who may be able to join
- 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
- Subjects older than 18 years (matched population for age and sex with ALS)
- Neurological testing and examination showing no neurological disorders.
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- Not having severe disease or life- functional prognosis
- 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
Who cannot join
- Subjects younger than 18 years
- Patient unable to provide informed consent
- Having severe disease or life- functional prognosis
- Contraindications MRI
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
France13 sites
CHU Pontchaillou
Angers
CHU Cote de Nacre
Caen
CHU Gabriel Montpied
Clermont-Ferrand
CHRU, Hôpital Salengro
Lille
Hôpital Dupuytren
Limoges
Hôpital Neurologique Pierre Wertheimer
Lyon
AP-HM,Hôpital de la Timone
Marseille
Hôpital Gui de Chauliac
Montpellier
Hôpital de l'Archet 1, CHU
Nice
Hôpital La Pitié (AP-HP)
Paris
Centre Hospitalier
Saint-Brieuc
Hôpital Nord
Saint-Etienne
CHU Bretonneau
Tours
About this study
Official title: Study of Predictive Factors of Progression of Motor Neurone Disease Prognosis and Endophenotype Biomarkers French Database Set up
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.
Read the detailed 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).
- Being tested
- biomarkers (composite analysis)
- Keywords
- multimodal biomarkers, prognosis, endophenotypes
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Motor Neuron Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
biomarkers (composite analysis)Other
biological, imaging, electrophysiological and anatomopathological examinations
| Study group | Role | What they receive |
|---|---|---|
| patients | follow up of patients with amyotrophic lateral sclerosis from the first signs to the end of the disease Receives: Other: biomarkers (composite analysis) | |
| neurological controls | patients with other neurological disease Receives: Other: biomarkers (composite analysis) | |
| healthy controls | age matched healthy controls Receives: Other: biomarkers (composite analysis) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change of biomarkers of survival
The predictive value of the clinical, biological, imaging, and electrophysiological biomarkers of survival will be analyzed according to the life duration (months) without events (i.e. severe comorbidity, 24 hours of non invasive ventilation, tracheotomy)
Measured: From date of randomization until the date of first documented progression , assessed up to 100 months
Secondary outcomes (3)
Change of biomarkers of disease progression
The predictive value of the clinical, biological, imaging, and electrophysiological biomarkers of prognosis will be analyzed according to the rate of progression of the ALSFRS-R score
Measured: baseline, 3 months, 6 months, 9 months, 12 months, 15 months, 24 months, 36 months, 48 months (average)
Clinical endophenotypes according to the survival duration
The determination of the different clinical, biological, radiological and electrophysiological endophenotypes (clusters of the same characteristics) according to the survival duration
Measured: From date of randomization until the date of death related with ALS or tracheotomy or continuous non invasive ventilation (24 hours a day), whichever came first, assessed up to 100 months
Genetic endophenotypes
the determination of the different clinical, biological, radiological and electrophysiological endophenotypes (clusters of the same characteristics) according to the according to the genetic forms (SOD1, TDP43, FUS, C9orf72,...)
Measured: the date of death related with ALS or tracheotomy or continuous non invasive ventilation (24 hours a day), whichever came first, assessed up to 100 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2015 (estimated)
- Study start12 Jan 2015
- Primary completion30 Jun 2025
- Study completion30 Jun 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by University Hospital, LilleThe 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
- 500 participants (actual)
- Target duration
- 10 Years
Sponsor and investigators
- Lead sponsor
- University Hospital, Lille
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- David Devos, MD, PhD, Study chair, University Hospital, Lille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02360891
- Sponsor's protocol ID
- 2012-50
- Other IDs
- 2013-A00969-36
- Acronym
- PULSE
- Status last verified
- Apr 2026
- First submitted
- 7 Jan 2015
- Last updated
- 5 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Grolez G, Kyheng M, Lopes R, Moreau C, Timmerman K, Auger F, Kuchcinski G, Duhamel A, Jissendi-Tchofo P, Besson P, Laloux C, Petrault M, Devedjian JC, Perez T, Pradat PF, Defebvre L, Bordet R, Danel-Brunaud V, Devos D. MRI of the cervical spinal cord predicts respiratory dysfunction in ALS. Sci Rep. 2018 Jan 29;8(1):1828. doi: 10.1038/s41598-018-19938-2.
PubMed 29379040Grapperon AM, Harlay V, Boucekine M, Devos D, Rolland AS, Desnuelle C, Delmont E, Verschueren A, Attarian S. Could the motor unit number index be an early prognostic biomarker for amyotrophic lateral sclerosis? Clin Neurophysiol. 2024 Jul;163:47-55. doi: 10.1016/j.clinph.2024.04.013. Epub 2024 Apr 26.
PubMed 38703699Le Gall L, Duddy WJ, Martinat C, Mariot V, Connolly O, Milla V, Anakor E, Ouandaogo ZG, Millecamps S, Laine J, Vijayakumar UG, Knoblach S, Raoul C, Lucas O, Loeffler JP, Bede P, Behin A, Blasco H, Bruneteau G, Del Mar Amador M, Devos D, Henriques A, Hesters A, Lacomblez L, Laforet P, Langlet T, Leblanc P, Le Forestier N, Maisonobe T, Meininger V, Robelin L, Salachas F, Stojkovic T, Querin G, Dumonceaux J, Butler Browne G, Gonzalez De Aguilar JL, Duguez S, Pradat PF. Muscle cells of sporadic amyotrophic lateral sclerosis patients secrete neurotoxic vesicles. J Cachexia Sarcopenia Muscle. 2022 Apr;13(2):1385-1402. doi: 10.1002/jcsm.12945. Epub 2022 Feb 22.
PubMed 35194965
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Predictive Factors of Progression of Motor Neurone Disease Prognosis and Endophenotype Biomarkers French Database Set up
- ClinicalTrials.gov ID
- NCT02360891
- Acronym / sponsor ID
- PULSE / 2012-50
- Keywords
- multimodal biomarkers, prognosis, endophenotypes
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 500 participants (actual)
- Lead sponsor
- University Hospital, LilleUniversity, hospital or other
- Study start
- 12 Jan 2015 (actual)
- Primary completion
- 30 Jun 2025 (actual)
- Study completion
- 30 Jun 2025 (actual)
- First posted
- 11 Feb 2015 (estimated)
- 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.
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.
Detailed 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).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 10 Years
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| patients | – | Other: biomarkers (composite analysis)follow up of patients with amyotrophic lateral sclerosis from the first signs to the end of the disease |
| neurological controls | – | Other: biomarkers (composite analysis)patients with other neurological disease |
| healthy controls | – | Other: biomarkers (composite analysis)age matched healthy controls |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- Multicentric French cohort of 1000 ALS patients, 100 neurological controls and 200 healthy controls
Key inclusion criteria (11 listed)
- 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
- Subjects older than 18 years (matched population for age and sex with ALS)
Key exclusion criteria (4 listed)
- Subjects younger than 18 years
- Patient unable to provide informed consent
- Having severe disease or life- functional prognosis
- Contraindications MRI
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 |
|---|---|---|
| biomarkers (composite analysis)biological, imaging, electrophysiological and anatomopathological examinations | Other | healthy controls, neurological controls, patients |
Primary outcome
Change of biomarkers of survival
Time frame: From date of randomization until the date of first documented progression , assessed up to 100 months
The predictive value of the clinical, biological, imaging, and electrophysiological biomarkers of survival will be analyzed according to the life duration (months) without events (i.e. severe comorbidity, 24 hours of non invasive ventilation, tracheotomy)
3 secondary outcomes
Change of biomarkers of disease progression
Time frame: baseline, 3 months, 6 months, 9 months, 12 months, 15 months, 24 months, 36 months, 48 months (average)
Clinical endophenotypes according to the survival duration
Time frame: From date of randomization until the date of death related with ALS or tracheotomy or continuous non invasive ventilation (24 hours a day), whichever came first, assessed up to 100 months
Genetic endophenotypes
Time frame: the date of death related with ALS or tracheotomy or continuous non invasive ventilation (24 hours a day), whichever came first, assessed up to 100 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2026.
- Results publication: PubMed 29379040Grolez G, Kyheng M, Lopes R, Moreau C, Timmerman K, Auger F, Kuchcinski G, Duhamel A, Jissendi-Tchofo P, Besson P, Laloux C, Petrault M, Devedjian JC, Perez T, Pradat PF, Defebvre L, Bordet R, Danel-Brunaud V, Devos D. MRI of the cervical spinal cord predicts respiratory dysfunction in ALS. Sci Rep. 2018 Jan 29;8(1):1828. doi: 10.1038/s41598-018-19938-2.
- Derived from this study: PubMed 38703699Grapperon AM, Harlay V, Boucekine M, Devos D, Rolland AS, Desnuelle C, Delmont E, Verschueren A, Attarian S. Could the motor unit number index be an early prognostic biomarker for amyotrophic lateral sclerosis? Clin Neurophysiol. 2024 Jul;163:47-55. doi: 10.1016/j.clinph.2024.04.013. Epub 2024 Apr 26.
- Derived from this study: PubMed 35194965Le Gall L, Duddy WJ, Martinat C, Mariot V, Connolly O, Milla V, Anakor E, Ouandaogo ZG, Millecamps S, Laine J, Vijayakumar UG, Knoblach S, Raoul C, Lucas O, Loeffler JP, Bede P, Behin A, Blasco H, Bruneteau G, Del Mar Amador M, Devos D, Henriques A, Hesters A, Lacomblez L, Laforet P, Langlet T, Leblanc P, Le Forestier N, Maisonobe T, Meininger V, Robelin L, Salachas F, Stojkovic T, Querin G, Dumonceaux J, Butler Browne G, Gonzalez De Aguilar JL, Duguez S, Pradat PF. Muscle cells of sporadic amyotrophic lateral sclerosis patients secrete neurotoxic vesicles. J Cachexia Sarcopenia Muscle. 2022 Apr;13(2):1385-1402. doi: 10.1002/jcsm.12945. Epub 2022 Feb 22.
- Individual participant dataSharing planned: No
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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.
David Devos, MD, PhD study chair
University Hospital, Lille
13 sites in 1 country: 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 University Hospital, Lille at 13 sites in France.
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 11 inclusion and 4 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: finished, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in France. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change of biomarkers of survival (time frame: From date of randomization until the date of first documented progression , assessed up to 100 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02360891, which also lists 3 linked publications.
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 5 May 2026.