Clinical trial NCT06578195: ASSESS ALL ALS Study
Official title: ASSESS ALL ALS - Longitudinal Biomarker Study for Symptomatic ALS and Control Participants
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Amyotrophic Lateral Sclerosis
biomarker
observational
Lead sponsor: St. Joseph's Hospital and Medical Center, Phoenix
Sponsor class: OTHER
Collaborators: name: Massachusetts General Hospital
name: National Institute of Neurological Disorders and Stroke (NINDS)
name: Foundation for the National Institutes of Health
First posted: 2024-08-29
Enrollment: 2000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: An initial cohort of approximately 2000 to 3000 participants will be recruited for this study. This includes at least 1600 symptomatic ALS participants (Cohort 1) and at least 450 control participants (Cohort 2). Though the exact percentages of in-clinic and remote participants are unknown at this time, it is anticipated that approximately 600 of the symptomatic ALS participants and 450 of the control participants will be recruited from the site clinic or local ALS community and will be coming to the sites for study visits. These numbers may differ substantially from the final enrollment numbers based on preference of participants enrolling in the study.

Brief summary: The ALL ALS Clinical Research Consortium is establishing research to collect a wide range of samples, clinical information and measurements from Amyotrophic Lateral Sclerosis (ALS) symptomatic, ALS gene carriers and control cohorts. This consortium is being funded by the National Institutes of Health/National Institute of Neurological Disorders and Stroke (NIH/NINDS) and managed by two clinical coordinating centers (CCC) at Barrow Neurological Institute and Massachusetts General Hospital. The clinical sites are distributed across the country, and led by a group of collaborative principal investigators. Once data and samples are collected and harmonized, it will be made available to research community for future research into ALS and related neurological diseases.

ASSESS protocol is specific for symptomatic ALS and control participants. This protocol includes both on-site and off-site(remote) participants. The participants will be followed for 24 months (2 years), and will include collection of medical history, clinical outcomes, and blood samples once in 4 months. Additionally, the participants will complete patient reported outcomes and speech recordings once a month. Participants who are coming into clinic may also provide optional Cerebrospinal Fluid (CSF) samples.


Eligibility criteria: Inclusion Criteria for ALS participants:

1. Age 18 years or older
2. Capable of providing informed consent
3. Willing to follow study procedures
4. Diagnosis of ALS by a physician
5. Access to a smartphone, computer or tablet, and internet (need not be in the home - access to a public library or other available computer with internet connection is sufficient)

Inclusion Criteria for control participants:

1. Age 18 years or older
2. Capable of providing informed consent
3. Willing to follow study procedures
4. No diagnosis of ALS , Progressive Muscular Atrophy (PMA) or Primary Lateral Sclerosis (PLS)
5. No history of familial ALS/Frontotemporal Dementia (FTD) in a close family member\*\* unless the participant has previously tested negative for the known causative ALS genes. Participants with a family history of singleton ALS are permitted to enroll.

   * \*\* Defined by the presence of a known ALS causative gene such as C9orf72 in a family member or a family history suggestive of an inherited ALS/FTD syndrome defined by two family members with a history of ALS and/or FTD.
6. Access to a smartphone, computer or tablet, and internet (need not be in the home - access to a public library or other available computer with internet connection is sufficient)

Exclusion Criteria for all participants:

1. Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, active suicidal ideation, suicide attempt, or untreated major depression \<= 90 days of screening, that would interfere with the study procedure, according to Investigator's judgement.
2. Clinically significant unstable medical condition (other than ALS) (e.g., cardiovascular instability, systemic infection, untreated thyroid dysfunction, malignant and potentially progressive cancer) that would render the participant unlikely to be able to complete 12 months of follow-up, according to Investigator's judgment.

Exclusion Criteria for participants undergoing optional Lumbar Puncture

1. Medically unable to undergo lumbar puncture (LP) as determined by the site investigator (i.e., bleeding disorder, a skin infection at or near the LP site, known or suspected intracranial or intraspinal tumor or other cause of increased intracranial pressure).
2. Allergy to Lidocaine or other local anesthetic agents.
3. Use of anticoagulant medication or antiplatelet medications (aside from aspirin 81 mg) that cannot be safely withheld prior to lumbar puncture.
4. Blood dyscrasia, abnormal bleeding diathesis, or the use of dialysis for renal failure.
5. Current pregnancy based on participant self-report
6. Clinical judgement of the site investigator that the participant would be unable to undergo multiple lumbar punctures.

Primary outcomes:
measure: ALS Functional Rating Scale-Revised (ALSFRS-R)
timeFrame: Baseline, 4, 8, 12, 16, 20 and 24 months for ALS participants only.
measure: ALS Functional Rating Scale-Revised Self entry (ALSFRS-RSE)
timeFrame: Baseline, 2, 4 ,6, 8, 10, 12, 14,16, 18,20, 22 and 24 months for ALS participants only
Secondary outcomes:
measure: ALS Impairment Multidomain scale (AIMS)
timeFrame: 1, 3, 5,7,9,11,13,15,17,19,21 and 23 months from baseline for ALS participants only
measure: Social Determinants of Health
timeFrame: Months 3 and 4 for all cohorts
measure: Environmental History
timeFrame: Month 1 for all cohorts
measure: Cognitive assessment (ECAS)
timeFrame: Baseline, 12 and 24 months for all cohorts
measure: Vital Capacity
timeFrame: Baseline, 4,8,12,16,20 and 24 months for on-site ALS participants; Baseline, 12 and 24 months for control participants
measure: Digital Speech Assessment
timeFrame: Baseline, and monthly (at month 1,2, 3 through month 24 from baseline)
measure: HHD of 3 hand muscles bilaterally
timeFrame: Baseline, 4,8,12,16,20 and 24 months for on-site ALS participants; Baseline, 12 and 24 months for control participants
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Barrow Neurological Institute
state: Arizona
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: La Jolla
country: United States
facility: University of California San Diego
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Aurora
country: United States
facility: University of Colorado Anschutz Medical Campus
state: Colorado
city: New Britain
country: United States
facility: Hospital For Special Care
state: Connecticut
city: Washington
country: United States
facility: Georgetown University
state: District of Columbia
city: Jacksonville
country: United States
facility: Mayo Clinic
state: Florida
city: Boise
country: United States
facility: Saint Alphonsus Regional Medical Center
state: Idaho
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University
state: Indiana
city: Baltimore
country: United States
facility: John Hopkins University
state: Maryland
city: Bethseda
country: United States
facility: Nih/Ninds
state: Maryland
city: Boston
country: United States
facility: Massachusetts General Brigham
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Detroit
country: United States
facility: Henry Ford Health
state: Michigan
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Omaha
country: United States
facility: University of Nebraska Medical Center
state: Nebraska
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: New York
country: United States
facility: Columbia University
state: New York
city: Durham
country: United States
facility: Duke University
state: North Carolina
city: Colombus
country: United States
facility: Ohio State University
state: Ohio
city: Portland
country: United States
facility: Providence ALS Center
state: Oregon
city: Hershey
country: United States
facility: Penn State Health
state: Pennsylvania
city: Philadelphia
country: United States
facility: Temple University
state: Pennsylvania
city: Dallas
country: United States
facility: Texas Neurology
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Seattle
country: United States
facility: University of Washington
state: Washington
city: San Juan
country: Puerto Rico
facility: CHALS-CCT, University of Puerto Rico, Medical Sciences Campus
Study officials:
References: