The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study
The investigators aim to recruit unaffected (healthy) people from families with a known genetic mutation in which at least two relatives have been affected with Amyotrophic Lateral Sclerosis (ALS). Our goal is to identify factors, both genetic and environmental, which put people at risk for developing ALS in the future.
Disease:Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS, Healthy Volunteer with a Family History of ALS
Study Type:Observational Study
Study Category:Biomarkers/Imaging , Epidemiology
Study Chair(s)/Principal Investigator(s):
Michael Benatar, MBChB, MS, DPhil (University of Miami)
Clinicaltrials.gov ID (11 digit #):NCT00317616
Coordinating Center Contact InformationUniversity of Miami Miller School of Medicine
Miami, Florida 33136 United States
Full Study Summary:
Healthy people from familial ALS families with a known genetic mutation will be included in this study. The investigators encourage people who know that they carry the mutation that affects their family as well as those who do not know their genetic status to contact us. Those who wish to participate and to learn the results of genetic testing, may do so after undergoing genetic counseling. It is also possible to participate without learning the results of genetic testing. Participants in the study will travel to Miami (at our expense) approximately every 12-24 months to complete study visits.
Study Sponsor:Muscular Dystrophy Association; ALS Association
Until appearance of manifest disease
Estimated Study Start Date:04/01/2006
Estimated Study Completion Date:12/31/2027
Posting Last Modified Date:01/27/2020
Date Study Added to alsconsortium.org:10/12/2011
Time since Symptom Onset:N/A
Time since Diagnosis:N/A
Can participants use Riluzole?Yes
People from anywhere in the US and Canada may participate. Costs of genetic testing and travel are covered by the study.
-A member of a family in which a mutation in a gene associated with ALS has been identified. This may include a family in which at least two relatives have been or currently are affected with ALS.
-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.
-Willingness to undergo genetic testing, with the option of whether or not to learn the results.
-Willingness to travel to Miami approximately every 12 to 24 months for in-person study evaluations.
-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.
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