Clinical trial NCT05204017: Comprehensive Analysis Platform To Understand, Remedy and Eliminate ALS
Official title: Comprehensive Analysis Platform To Understand, Remedy and Eliminate ALS
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Amyotrophic Lateral Sclerosis
Primary Lateral Sclerosis
Progressive Muscular Atrophy
Frontotemporal Degeneration
Keywords: Biomarker
Biorepository
ALS
Disease Progression
MRI
Biofluids
Lead sponsor: University of Alberta
Sponsor class: OTHER
Collaborators: name: McGill University
name: Laval University
name: University of Toronto
name: Simon Fraser University
First posted: 2022-01-24
Enrollment: 150
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: CASE_CONTROL
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: People living with ALS and related motor neuron diseases including ALS-Frontotemporal Dementia (ALS-FTD), Primary Lateral Sclerosis (PLS), Progressive Muscular Atrophy (PMA) and asymptomatic individuals with a known ALS mutation

AND

Healthy controls that are age and sex matched to patients

Brief summary: CAPTURE ALS is a long-term data and biorepository platform that will facilitate future ALS research. CAPTURE ALS will provide the standardized systems and tools necessary to collect, store, and analyze vast amounts of multimodal information about ALS. These multimodal datasets and biosamples will be made available for use by researchers or industry across Canada and around the world in accordance with the CAPTURE ALS Data Sharing Policy to advance research on ALS.

Detailed description: In this observational study, participants with ALS and related disorders will be followed longitudinally over the course of a year to examine disease progression. Participants with ALS or a related disorder will have up to 5 in-person visits at screening, 0, 4, 8 and 12 months. Healthy controls are included as comparisons and will have 3 in-person visits at screening, 0 and 8 months. At each visit after screening, patients and healthy controls will undergo an MRI, neurocognitive and speech testing, clinical evaluations specific to ALS (ALSFRS-R, FVC, neurological exam, which are not completed by controls), answer questionnaires about their health and have biospecimens, including blood, saliva and optionally cerebrospinal fluid, collected.

Eligibility criteria: \[PATIENTS\]

Inclusion Criteria:

1. Has ALS, classified as definite, probable, laboratory-supported probable, or possible ALS according to the revised El Escorial criteria or a related neurodegenerative disorder including ALS-FTD, PLS, PMA or asymptomatic individuals with a known ALS mutation (as previously confirmed during regular clinical care)
2. Be of the age of majority in their province of residence/treatment
3. Have the cognitive capacity to provide informed consent
4. Have proficiency in English or French in order to understand study instructions and respond to questionnaires

Exclusion Criteria:

N/A

\[HEALTHY CONTROLS\]

Inclusion Criteria:

1. Be between the ages of 40-80 unless age matched to a patient (+/-3 years) who is already enrolled
2. Be the age of majority in their province of residence/treatment
3. Have the cognitive capacity to provide informed consent
4. Have proficiency in English of French to understand study instructions and respond to questionnaires

Exclusion Criteria:

1. A history of a neurological disease, including Central Nervous System disease (e.g., stroke, head injury, epilepsy) or Peripheral Nervous System disease (neuropathy, myopathy)
2. A history of psychiatric disease (e.g. depression, bipolar disease) that is clinically diagnosed and/or with the current use of psychiatric medications (e.g. antidepressants) for an indication of a psychiatric disease.
3. Subjects ineligible for an MRI due to a pacemaker or other contraindication according to local MRI policies of the study centre.
4. Significant claustrophobia that would prohibit an MRI (subjects will be required to lie in an MRI scanner for approximately one hour).

Primary outcomes:
measure: ALS Functional Rating Scale-Revised (ALSFRS-R)
timeFrame: Once every 4 months for a year
measure: Forced Vital Capacity (FVC)
timeFrame: Once every 4 months for a year
measure: Speech Testing
timeFrame: Once every 4 months for a year
measure: Neurological dysfunction as detected during standard neurological examination
timeFrame: Once every 4 months for a year
measure: Magnetic Resonance Imaging (MRI)
timeFrame: Once every 4 months for a year
measure: Edinburgh Cognitive and Behavioural Screen (ECAS)
timeFrame: Once every 4 months for a year
measure: ALS Assessment Questionnaire (ALSAQ-5)
timeFrame: Once every 4 months for a year
measure: World Health Organization Quality of Life Questionnaire (WHO-QOL-BREF)
timeFrame: Once every 4 months for a year
Secondary outcomes:
Other outcomes:
Locations:
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Toronto
country: Canada
facility: University of Toronto / Sunnybrook Health Sciences Centre
state: Ontario
city: Montreal
country: Canada
facility: McGill University / Montreal Neurological Institute and Hospital
state: Quebec
city: Québec
country: Canada
facility: CHU de Quebec -Universite de Laval
state: Quebec
Study officials:
affiliation: University of Alberta
name: Sanjay Kalra, MD
role: PRINCIPAL_INVESTIGATOR
References: