Study of healthy individuals at genetic risk for ALS
Official title: The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study
Run by University of Miami · NCT00317616 · Data updated 2026-11-12
This study is looking at healthy people who have a family history of ALS to see how their genes and environment might affect their chances of developing the disease in the future.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 500 (estimated) |
| Sponsor | University of Miami Academic / other |
| Locations | 1 site (Miami, Florida) |
| Status | Recruiting |
| Started | April 2006 |
| Main results expected | December 2037 |
| Study ends | December 2037 |
| Registry record updated | March 20, 2026 |
| Trial ID | NCT00317616 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are healthy individuals from families with a known genetic mutation associated with ALS who have at least a 50% probability of carrying the mutation.
✕Probably not a fit
Patients who are already diagnosed with ALS or have conditions that could confound the study results will not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) study is an observational study aimed at understanding the natural history and biomarkers of individuals who are genetically predisposed to develop ALS but are currently healthy. Participants from familial ALS pedigrees with known genetic mutations will be recruited to undergo periodic assessments, including biomarker procedures and health evaluations, over a span of 10 years or more. The study will also explore genetic and environmental factors that may contribute to the onset of ALS. Participants will have the option to learn their genetic testing results after receiving genetic counseling.
Why it matters
Potential benefit: If successful, this study could lead to better understanding and early intervention strategies for individuals at risk of developing ALS.
How similar studies have gone: Other studies focusing on genetic predisposition to ALS have shown promise, but this specific approach to studying pre-symptomatic individuals is relatively novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria * A member of a family in which a mutation in a gene associated with ALS has been identified. * No symptoms to suggest the presence of ALS (i.e. study participants must currently be healthy). * Having at least 50% probability of carrying an ALS associated gene mutation based on family pedigree. * Willingness to undergo genetic testing, with the option of whether or not to learn the results. * Willingness and availability to travel to Miami for a few days approximately every 12 to 24 months to complete biomarker procedures (e.g, MRI, blood draws, spinal tab/lumbar puncture, cognitive testing) Exclusion Criteria * Diagnosis of ALS * Any condition or situation which, in the PI's opinion, could confound the biomarker data or may interfere with the individual's participation and compliance with the study protocol, including but not limited to neurological, psychological and/or medical conditions.
Where it's running
-
University of Miami
Miami, Florida, United States Recruiting
Study contacts
- Principal investigator: Michael G Benatar, MD, PhD. — University of Miami
- Study coordinator: Anne-Laure M Grignon, MD
- Email: fals@med.miami.edu
- Phone: 1-888-413-9315
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.