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

Study Purpose:

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 Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Michael Benatar, MBChB, MS, DPhil (University of Miami)

Clinicaltrials.gov ID (11 digit #):

NCT00317616

Neals Affiliated?

Yes

Coordinating Center Contact Information

University of Miami Miller School of Medicine
Anne-Laure Grignon / .(JavaScript must be enabled to view this email address) / 888-413-9315
Danielle Dauphin / .(JavaScript must be enabled to view this email address) 1120 NW 14 Street, STE 1312
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

Participant Duration:

Until appearance of manifest disease

Estimated Enrollment:

500

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

Eligibility Criteria

Gender:
Female, Male
Minimum Age:
18
Maximum Age:
N/A

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.

Inclusion Criteria
-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.

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.
Site Contact Information

University of Miami Miller School of Medicine
Anne-Laure Grignon / .(JavaScript must be enabled to view this email address) / 888-413-9315
1120 NW 14 Street, STE 1312
Miami, Florida 33136
United States