The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study (Pre-fALS)
This study is currently recruiting participants.
(see Contacts and Locations)
Verified July 2016 by University of Miami
Sponsor:
Michael Benatar
Collaborator:
ALS Association
Information provided by (Responsible Party):
Michael Benatar, University of Miami
ClinicalTrials.gov Identifier:
NCT00317616
First received: April 24, 2006
Last updated: July 18, 2016
Last verified: July 2016
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Purpose
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.
| Condition |
|---|
|
Amyotrophic Lateral Sclerosis |
| Study Type: | Observational |
| Study Design: | Observational Model: Cohort Time Perspective: Prospective |
| Official Title: | The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study |
Resource links provided by NLM:
Genetics Home Reference related topics:
amyotrophic lateral sclerosis
MedlinePlus related topics:
Amyotrophic Lateral Sclerosis
U.S. FDA Resources
Further study details as provided by University of Miami:
Primary Outcome Measures:
- Risk Factors for Progression to familial ALS [ Time Frame: Years ] [ Designated as safety issue: No ]
Biospecimen Retention: Samples With DNA
Serum, Plasma, CSF, DNA, Urine, Cell lines
| Estimated Enrollment: | 500 |
| Study Start Date: | April 2006 |
| Estimated Primary Completion Date: | December 2019 (Final data collection date for primary outcome measure) |
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 up to 10 years and will perform various biomarker procedures. Between visits, participants will complete phone calls about their health.
Eligibility| Ages Eligible for Study: | 18 Years and older (Adult, Senior) |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
Study Population
Healthy individuals who harbor a mutation in a gene associated with ALS.
Criteria
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.
Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study.
To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
For general information, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT00317616
Please refer to this study by its ClinicalTrials.gov identifier: NCT00317616
Contacts
| Contact: Eliana M Reyes | 1-888-413-9315 | fals@med.miami.edu | |
| Contact: Danielle Dauphin | 1-888-413-9315 |
Locations
| United States, Florida | |
| University of Miami | Recruiting |
| Miami, Florida, United States, 33136 | |
| Contact: Eliana Reyes 888-413-9315 fals@med.miami.edu | |
| Contact: Danielle Dauphin | |
Sponsors and Collaborators
Michael Benatar
ALS Association
Investigators
| Principal Investigator: | Michael G Benatar, MD, PhD. | University of Miami |
More Information
| Responsible Party: | Michael Benatar, MBChB, MS, DPhil, University of Miami |
| ClinicalTrials.gov Identifier: | NCT00317616 History of Changes |
| Other Study ID Numbers: | 1369-2005 |
| Study First Received: | April 24, 2006 |
| Last Updated: | July 18, 2016 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by University of Miami:
|
FALS SOD1 familial genetic testing |
genetic counseling ALS C9ORF72 |
Additional relevant MeSH terms:
|
Sclerosis Motor Neuron Disease Amyotrophic Lateral Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases |
Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases |
ClinicalTrials.gov processed this record on September 27, 2016