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The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study

Who can join

Ages 18 and older · All sexes

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.

About the study

Pre-fALS is a prospective natural history and biomarker study of people not yet affected with ALS, but who are at genetic risk for developing ALS. The investigators aim to recruit unaffected (healthy) people from familial ALS (fALS) pedigrees in which a known genetic mutation associated with ALS has been identified; for this study, a fALS pedigree is one with two biologically related individuals who have or have had ALS and/or FTD. Individuals who may be at genetic risk for ALS and who belong to families with at least one affected family member who has tested positive for a known ALS genetic mutation may also be eligible to participate. Our goal is to study the pre-symptomatic phase, onset and progression of ALS and to learn more about genetic and environmental factors that put people at risk for developing ALS.

Sponsor: University of Miami · Participants: 500 · Started: 2006-04

Contact the study team

• Anne-Laure M Grignon, MD · Phone: 1-888-413-9315

Official record on ClinicalTrials.gov — NCT00317616

Locations in the U.S.

Conditions

• Amyotrophic Lateral Sclerosis

From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.