The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study (Pre-fALS)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT00317616 |
Recruitment Status :
Recruiting
First Posted : April 25, 2006
Last Update Posted : April 13, 2022
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Condition or disease |
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Amyotrophic Lateral Sclerosis |
Study Type : | Observational |
Estimated Enrollment : | 500 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official Title: | The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study |
Study Start Date : | April 2006 |
Estimated Primary Completion Date : | December 2027 |
Estimated Study Completion Date : | December 2027 |

Group/Cohort |
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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).
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- Risk Factors for Progression to familial ALS [ Time Frame: Years ]
Biospecimen Retention: Samples With DNA

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00317616
Contact: Anne-Laure M Grignon, MD | 1-888-413-9315 | fals@med.miami.edu |
United States, Florida | |
University of Miami | Recruiting |
Miami, Florida, United States, 33136 | |
Contact: Anne-Laure Grignon 888-413-9315 fals@med.miami.edu | |
Contact: Danielle Dauphin |
Principal Investigator: | Michael G Benatar, MD, PhD. | University of Miami |
Responsible Party: | Michael Benatar, Professor, University of Miami |
ClinicalTrials.gov Identifier: | NCT00317616 |
Other Study ID Numbers: |
Pre-fALS |
First Posted: | April 25, 2006 Key Record Dates |
Last Update Posted: | April 13, 2022 |
Last Verified: | April 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
FALS SOD1 familial genetic testing genetic counseling ALS |
C9ORF72 TARDBP VCP FUS PFN1 |
Motor Neuron Disease Amyotrophic Lateral Sclerosis Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases |
Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases |