PREVENT ALL ALS Study
ConditionAmyotrophic Lateral Sclerosis
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorSt. Joseph's Hospital and Medical Center, Phoenix
About this trial
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
This trial accepts healthy volunteersQualifiers
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.
Disqualifiers
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
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).
Trial population
Approximately 600 participants are planned for this study enrolled. They are aged 18 years or older, and are at risk of developing ALS.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
ALS Symptoms Questionnaire
Questions to identify development of symptoms related to ALS
Time frame
Baseline, 4, 8, 12,16,20, 24, 28, 32 and 36 months form baselineSecondary outcomes
1
Environmental History
Questionnaires completed by participants that includes questions about non-medical factors that affect health outcomes
Time frame
Once at Month 1 from Baseline2
Cognitive assessment (ECAS)
ECAS is a multi-domain assessment administered by study staff, to evaluate different aspects of cognitive function
Time frame
at Baseline, 12, 24 and 36 months from baseline3
Cognitive assessment (CDR-FTLD)
CDR-FTLD is a global assessment scale that evaluates severity of impairment across multiple domains in patients with Frontotemporal dementia (FTLD) spectrum disorders.
Time frame
at Baseline, 12, 24 and 36 months from baseline4
Hand-Held Dynamometry (HHD)
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.
Time frame
at Baseline, 12, 24 and 36 months from baselineSponsors and contacts
Click on the lead sponsor to view all of their trials.
St. Joseph's Hospital and Medical Center, Phoenix
Lead sponsor
Massachusetts General Hospital
Collaborator
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborator
Foundation for the National Institutes of Health
Collaborator
Source ClinicalTrials.gov