Research on familial ALS and genetic factors
Official title: Amyotrophic Lateral Sclerosis (ALS) Families Project
Run by Columbia University · NCT03865420 · Data updated 2026-11-12
This study is trying to understand how familial ALS develops by looking at family members of those affected and offering genetic counseling to help them learn about their risk and possibly find new ways to prevent or treat the disease.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 18 Years to 105 Years |
| Sex | All |
| Enrollment | 300 (estimated) |
| Sponsor | Columbia University Academic / other |
| Locations | 1 site (New York, New York) |
| Status | Recruiting |
| Started | September 11, 2018 |
| Main results expected | January 2027 |
| Study ends | January 2027 |
| Registry record updated | January 7, 2026 |
| Trial ID | NCT03865420 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include adults aged 18 or older with a family history of ALS or frontotemporal dementia, particularly those who are unaffected carriers of ALS-associated gene mutations.
✕Probably not a fit
Patients who are symptomatic for ALS or frontotemporal dementia will not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
The ALS Families Project invites family members of individuals with familial ALS to participate in research aimed at understanding the onset and progression of motor neuron degeneration. This observational study provides genetic counseling and testing to help participants assess their risk and decide whether to learn their genetic status. Participants, who are unaffected carriers of ALS/FTD-associated gene mutations, will be followed annually to gather crucial data that may lead to new therapies for ALS prevention and treatment. The study focuses on identifying early disease targets and intervention points.
Why it matters
Potential benefit: If successful, this research could lead to breakthroughs in preventing or slowing the progression of ALS in at-risk individuals.
How similar studies have gone: Other studies focusing on genetic factors in familial ALS have shown promise, indicating that this approach is both relevant and potentially impactful.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Men or women of any race or ethnicity aged 18 or older * No symptoms of ALS or fronto-temporal dementia at enrollment * Scenario 1: has already had genetic testing that identified an ALS-spectrum gene mutation. * Scenario 2: has a first degree relative who was/is an obligate carrier of a familial ALS-spectrum gene mutation. * Scenario 3: has a first degree relative who has/had an ALS-spectrum diagnosis who had a confirmed ALS-spectrum gene mutation or comes from a family with a high burden of ALS-spectrum diagnoses and a known ALS-spectrum gene mutation. * Scenario 4: is deemed to be at high risk for carrying an ALS-spectrum gene mutation as judged by a review of the family structure and genetic information by the study team. * Willing to undergo genetic analysis, 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 for participation in biosample portion of the study: * Known HIV * Known hepatitis B * Known hepatitis C
Where it's running
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Columbia University
New York, New York, United States Recruiting
Study contacts
- Principal investigator: Matthew Harms, MD — Columbia University
- Study coordinator: Elizabeth Harrington, MS, CGC
- Email: ALSFamiliesProject@cumc.columbia.edu
- Phone: 347-852-5315
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.