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 : September 19, 2022
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Condition or disease |
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ALS |
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 |
Observational Model: | Family-Based |
Time Perspective: | Prospective |
Official Title: | Amyotrophic Lateral Sclerosis (ALS) Families Project |
Actual Study Start Date : | September 11, 2018 |
Estimated Primary Completion Date : | September 2023 |
Estimated Study Completion Date : | September 2023 |

- 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

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, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years to 105 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
Inclusion Criteria:
- Men or women of any race or ethnicity aged 18 or older
- No symptoms of ALS or fronto-temporal dementia at enrollment
- Have a first degree relative who had an ALS-spectrum diagnosis with a confirmed ALS-spectrum gene mutation; or already have had genetic testing and have tested positive for an ALS-spectrum gene mutation.
- Willing to undergo genetic testing with option of whether or not to learn results
- Willing to travel to Columbia University Irving Medical Center (CUIMC) every 6-24 months for study procedures
- Capable of providing informed consent and following study procedures, or has a legally authorized representative who is able to consent for the subject.
Exclusion Criteria:
- Known HIV
- Known hepatitis B
- Known hepatitis C

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 | |
Columbia University | Recruiting |
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 |
Responsible Party: | Columbia University |
ClinicalTrials.gov Identifier: | NCT03865420 |
Other Study ID Numbers: |
AAAR7275 |
First Posted: | March 6, 2019 Key Record Dates |
Last Update Posted: | September 19, 2022 |
Last Verified: | September 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 |
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