PREVENT ALL ALS Study
- Conditions
- Amyotrophic Lateral Sclerosis
- Registration Number
- NCT06581861
- Lead Sponsor
- St. Joseph's Hospital and Medical Center, Phoenix
- 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 begin 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.
PREVENT protocol is specific for asymptomatic participants who are genetically at risk for ALS. The participants will be followed for up to 36 months (3 years), and will include 4 in-person on-site visits once a year and 6 off-site(remote) visits once in 4 months. The study includes 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 in 4 months. Participants may also provide optional Cerebrospinal Fluid (CSF) samples.The participants may also opt into a sub-study if they are interested in genetic testing for ALS causative genes. The sub-study will involve a minimum of 3 visits over a course of 2-3 months. This will include a screening/pre-test genetic counseling visit, a return of genetic results and a post-test counseling visit.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 600
- Age 18 years or older
- Capable of providing informed consent
- Willing to follow study procedures
- First-degree relative of a known carrier of any ALS causative gene1 (regardless of whether ALS or FTD has actually been symptomatic in the family) OR First-degree relative of an individual with ALS and/or FTD in a family with a "compelling family history" of ALS/FTD, regardless of whether genetic testing has occurred in symptomatic family members. A "compelling family history" is defined as a pedigree with at least 2 close relatives who had ALS or FTD, with at least one of those family members having had ALS.
- 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)
- Evidence of neurological signs or symptoms concerning for ALS of FTD, at the discretion of the site investigator which will be communicated to the applicant along with referral for appropriate clinical follow-up.
- Significant cognitive impairment, clinical dementia, or unstable psychiatric illness, including psychosis, active suicidal ideation, suicide attempt, or untreated major depression <= 90 days (about 3 months) of screening, which in the opinion of the Investigator would interfere with the study procedures
- Clinically significant, unstable medical condition (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
- 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).
- Allergy to Lidocaine or other local anesthetic agents.
- Use of anticoagulant medication or antiplatelet medications (aside from aspirin 81 mg) that cannot be safely withheld prior to lumbar puncture.
- Blood dyscrasia, abnormal bleeding diathesis, or the use of dialysis for renal failure.
- Current pregnancy based on participant self-report
- Clinical judgement of the site investigator that the participant would be unable to undergo multiple lumbar punctures.
Inclusion Criteria for Genetic Testing Results Sub-study
- Age 18 years of age or older
- Capable of providing informed consent
- Willing to follow study procedures
- Currently enrolled in the PREVENT ALS Study
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method ALS Symptoms Questionnaire Baseline, 4, 8, 12,16,20, 24, 28, 32 and 36 months form baseline Questions to identify development of symptoms related to ALS
- Secondary Outcome Measures
Name Time Method Environmental History Once at Month 1 from Baseline Questionnaires completed by participants that includes questions about non-medical factors that affect health outcomes
Cognitive assessment (ECAS) at Baseline, 12, 24 and 36 months from baseline ECAS is a multi-domain assessment administered by study staff, to evaluate different aspects of cognitive function
Cognitive assessment (CDR-FTLD) at Baseline, 12, 24 and 36 months from baseline CDR-FTLD is a global assessment scale that evaluates severity of impairment across multiple domains in patients with Frontotemporal dementia (FTLD) spectrum disorders.
Hand-Held Dynamometry (HHD) at Baseline, 12, 24 and 36 months from baseline HHD is used as a quantitative measure of muscle strength. This will involve testing of the bilateral deltoid, biceps, triceps, wrist extension, FDI, ADM, APB, hip extension, quadriceps, hamstring, TA and grip strength.
Digital Speech Assessment at Baseline, and at months 4,8,12,16,20,24,28,32 and 36 months Participants will be asked to record voice samples at home once a month using a digital application installed on their device.
Vital Capacity (VC) at Baseline, 12, 24 and 36 months from baseline It is the change in respiratory function over time as measured by Slow Vital Capacity (SVC).
Trial Locations
- Locations (32)
Hospital for Special Care
🇺🇸New Britain, Connecticut, United States
Georgetown University
🇺🇸Washington, District of Columbia, United States
University of California San Diego
🇺🇸La Jolla, California, United States
Mayo Clinic
🇺🇸Jacksonville, Florida, United States
Nih/Ninds
🇺🇸Bethesda, Maryland, United States
University of Minnesota
🇺🇸Minneapolis, Minnesota, United States
Providence ALS Center
🇺🇸Portland, Oregon, United States
University of Alabama Birmingham
🇺🇸Birmingham, Alabama, United States
Barrow Neurological Institute
🇺🇸Phoenix, Arizona, United States
University of California Irvine
🇺🇸Orange, California, United States
University of California, San Francisco
🇺🇸San Francisco, California, United States
University of Colorado Anschutz Medical Campus
🇺🇸Aurora, Colorado, United States
Saint Alphonsus Regional Medical Center
🇺🇸Boise, Idaho, United States
Northwestern University
🇺🇸Chicago, Illinois, United States
Indiana University
🇺🇸Indianapolis, Indiana, United States
John Hopkins University
🇺🇸Baltimore, Maryland, United States
Massachusetts General Brigham
🇺🇸Boston, Massachusetts, United States
University of Michigan
🇺🇸Ann Arbor, Michigan, United States
Henry Ford Health
🇺🇸Detroit, Michigan, United States
Washington University
🇺🇸Saint Louis, Missouri, United States
University of Nebraska Medical Center
🇺🇸Omaha, Nebraska, United States
Dartmouth Hitchcock Medical Center
🇺🇸Lebanon, New Hampshire, United States
Columbia University
🇺🇸New York, New York, United States
Duke University
🇺🇸Durham, North Carolina, United States
Ohio State University
🇺🇸Columbus, Ohio, United States
Penn State Health
🇺🇸Hershey, Pennsylvania, United States
Temple University
🇺🇸Philadelphia, Pennsylvania, United States
Texas Neurology
🇺🇸Dallas, Texas, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
Virginia Commonwealth University
🇺🇸Richmond, Virginia, United States
University of Washington
🇺🇸Seattle, Washington, United States
CHALS-CCT, University of Puerto Rico, Medical Sciences Campus
🇵🇷San Juan, Puerto Rico