The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study
| Status: | Recruiting |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 18 - Any |
| Updated: | 1/17/2019 |
| Start Date: | April 2006 |
| End Date: | December 2027 |
| Contact: | Anne-Laure M Grignon, MD |
| Email: | fals@med.miami.edu |
| Phone: | 1-888-413-9315 |
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.
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.
Healthy individuals from fALS families with a known genetic mutation will be included in this
study. We encourage people who have previously undergone genetic testing and were found to
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 eligible to complete study
visits will travel to Miami (at our expense) approximately every 12-24 months for a period of
10 years or longer and will perform various biomarker procedures. Between visits,
participants will complete phone calls about their health.
study. We encourage people who have previously undergone genetic testing and were found to
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 eligible to complete study
visits will travel to Miami (at our expense) approximately every 12-24 months for a period of
10 years or longer and will perform various biomarker procedures. Between visits,
participants will complete phone calls about their health.
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.
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