Amyotrophic Lateral Sclerosis (ALS) Families Project
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|ClinicalTrials.gov Identifier: NCT03865420|
Recruitment Status : Recruiting
First Posted : March 6, 2019
Last Update Posted : October 12, 2020
|Condition or disease|
Approximately 10% of people with amyotrophic lateral sclerosis (ALS), or Lou Gehrig's Disease, have a family history of ALS or a related condition called frontotemporal dementia (FTD). In most of these familial cases, and a significant number of "sporadic" patients with no family history, a mutation is present in one of a growing number of genes that have been associated with ALS and/or FTD.
The ALS Families Project will study unaffected carriers of ALS/FTD-associated gene mutations to investigate the first steps in the disease process that leads to motor neuron degeneration, with the goal of identifying early disease targets and points of intervention to slow or stop disease onset and progression.
Unaffected individuals who have either a family member with a known ALS/FTD-associated gene mutation or have a strong family history of ALS and FTD are invited to participate in the ALS Families Project. For those who enroll, research visits will occur every 6-12 months.
|Study Type :||Observational|
|Estimated Enrollment :||60 participants|
|Official Title:||Amyotrophic Lateral Sclerosis (ALS) Families Project|
|Actual Study Start Date :||September 11, 2018|
|Estimated Primary Completion Date :||September 2021|
|Estimated Study Completion Date :||September 2022|
- Time to emergence of symptoms attributable to gene mutations [ Time Frame: Up to 3 years ]Emergence of symptoms will defined by the development of any of the following: a) any weakness on neurological examination, b) evidence of nerve loss on electromyography (EMG)-nerve conduction studies, or or c) evidence of cognitive impairment on the ALS-Cognitive Behavioral Scale (ALS-CBS).
Biospecimen Retention: Samples With DNA
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): NCT03865420
|Contact: Elizabeth Harrington, MS, CGC||347-852-5315||ALSFamiliesProject@cumc.columbia.edu|
|Contact: Matthew Harms, MD|
|United States, New York|
|New York, New York, United States, 10032|
|Contact: Elizabeth Harrington, MS, CGC 347-852-5315 ALSFamiliesProject@cumc.columbia.edu|
|Principal Investigator:||Matthew Harms, MD||Columbia University|