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PREVENT ALL ALS Study

NCT06581861 · St. Joseph's Hospital and Medical Center, Phoenix
In plain English

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Official title
PREVENT ALL ALS - Longitudinal Biomarker Study for Participants Who Are Genetically at Risk for Amyotrophic Lateral Sclerosis (ALS)
About this study
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.
Eligibility criteria
Inclusion Criteria: 1. Age 18 years or older 2. Capable of providing informed consent 3. Willing to follow study procedures 4. 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. 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) Exclusion Criteria: 1. 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. 2. 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 3. 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 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. Inclusion Criteria for Genetic Testing Results Sub-study 1. Age 18 years of age or older 2. Capable of providing informed consent 3. Willing to follow study procedures 4. Currently enrolled in the PREVENT ALS Study
Study design
Enrollment target: 600 participants
Age groups: adult, older_adult
Timeline
Starts: 2024-07-25
Estimated completion: 2029-07-25
Last updated: 2026-01-12
Primary outcomes
  • ALS Symptoms Questionnaire (Baseline, 4, 8, 12,16,20, 24, 28, 32 and 36 months form baseline)
Sponsor
St. Joseph's Hospital and Medical Center, Phoenix · other
With: Massachusetts General Hospital, National Institute of Neurological Disorders and Stroke (NINDS), Foundation for the National Institutes of Health
Contacts & investigators
ContactALL ALS Patient Navigator · contact · info@all-als.org · 602-845-0248
All locations (32)
University of Alabama BirminghamRecruiting
Birmingham, Alabama, United States
Barrow Neurological InstituteRecruiting
Phoenix, Arizona, United States
University of California San DiegoRecruiting
La Jolla, California, United States
University of California IrvineRecruiting
Orange, California, United States
University of California, San FranciscoRecruiting
San Francisco, California, United States
University of Colorado Anschutz Medical CampusRecruiting
Aurora, Colorado, United States
Hospital for Special CareRecruiting
New Britain, Connecticut, United States
Georgetown UniversityRecruiting
Washington D.C., District of Columbia, United States
Mayo ClinicRecruiting
Jacksonville, Florida, United States
Saint Alphonsus Regional Medical CenterRecruiting
Boise, Idaho, United States
Northwestern UniversityRecruiting
Chicago, Illinois, United States
Indiana UniversityRecruiting
Indianapolis, Indiana, United States
John Hopkins UniversityRecruiting
Baltimore, Maryland, United States
Nih/NindsRecruiting
Bethesda, Maryland, United States
Massachusetts General BrighamRecruiting
Boston, Massachusetts, United States
University of MichiganRecruiting
Ann Arbor, Michigan, United States
Henry Ford HealthRecruiting
Detroit, Michigan, United States
University of MinnesotaRecruiting
Minneapolis, Minnesota, United States
Washington UniversityRecruiting
St Louis, Missouri, United States
University of Nebraska Medical CenterRecruiting
Omaha, Nebraska, United States
Dartmouth Hitchcock Medical CenterRecruiting
Lebanon, New Hampshire, United States
Columbia UniversityRecruiting
New York, New York, United States
Duke UniversityRecruiting
Durham, North Carolina, United States
Ohio State UniversityRecruiting
Columbus, Ohio, United States
Providence ALS CenterRecruiting
Portland, Oregon, United States
Penn State HealthRecruiting
Hershey, Pennsylvania, United States
Temple UniversityRecruiting
Philadelphia, Pennsylvania, United States
Texas NeurologyRecruiting
Dallas, Texas, United States
University of UtahRecruiting
Salt Lake City, Utah, United States
Virginia Commonwealth UniversityRecruiting
Richmond, Virginia, United States
University of WashingtonRecruiting
Seattle, Washington, United States
CHALS-CCT, University of Puerto Rico, Medical Sciences CampusRecruiting
San Juan, Puerto Rico, Puerto Rico
PREVENT ALL ALS Study · TrialPath