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

Status: Recruiting
Location: See location...
Study Type: Observational
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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)

Locations
United States
Florida
University of Miami
RECRUITING
Miami
Contact Information
Primary
Anne-Laure M Grignon, MD
fals@med.miami.edu
1-888-413-9315
Time Frame
Start Date: 2006-04
Estimated Completion Date: 2037-12
Participants
Target number of participants: 500
Treatments
Unaffected individuals from families in which the genetic cause of ALS is known
This population would include pre-symptomatic individuals at genetic risk for ALS or a related neurodegenerative disorder (i.e., FTD).
Sponsors
Collaborators: ALS Association
Leads: University of Miami

This content was sourced from clinicaltrials.gov