Early detection and prevention for families with ALS/FTD risk
Pre-Symptomatic Familial ALS (Pre-fALS) Study: From Prodrome and Biomarkers to ALS/FTD Prevention
['FUNDING_OTHER'] · UNIVERSITY OF MIAMI SCHOOL OF MEDICINE · NIH-11135795
This project looks for early warning signs and blood markers in people who carry genetic changes that raise their chance of developing ALS or frontotemporal dementia so treatments can start before symptoms appear.
Quick facts
| Phase | ['FUNDING_OTHER'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF MIAMI SCHOOL OF MEDICINE (nih funded) |
| Locations | 1 site (CORAL GABLES, UNITED STATES) |
| Trial ID | NIH-11135795 on ClinicalTrials.gov |
What this research studies
You would be followed over time to track any early physical, behavioral, or biochemical changes before symptoms start, with regular visits, exams, and blood and other sample collection. The team measures markers such as neurofilament light chain and other lab and clinical tests to find signals that predict who will develop symptoms. They have developed criteria for a mild prodromal syndrome (mild motor impairment) and use these findings to identify people who might be eligible for prevention trials. The program also partners with industry to move promising prevention approaches into trials for specific genetic groups like SOD1 carriers.
Who could benefit from this research
Good fit: People who carry known pathogenic genetic variants linked to familial ALS or ALS/FTD (for example SOD1 or C9ORF72 carriers) but who do not yet have symptoms are the ideal candidates.
Not a fit: People without known high-risk genetic mutations or those who already have clear, symptomatic ALS/FTD are unlikely to gain from the pre-symptomatic focus of this program.
Why it matters
Potential benefit: If successful, the work could allow treatments to begin earlier or prevent ALS/FTD from developing in high-risk people.
How similar studies have performed: Pre-fALS has already shown that neurofilament light chain predicts conversion to clinical ALS, defined a prodromal mild motor impairment syndrome, and supported the launch of a prevention trial in SOD1 carriers.
Where this research is happening
CORAL GABLES, UNITED STATES
- UNIVERSITY OF MIAMI SCHOOL OF MEDICINE — CORAL GABLES, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: BENATAR, MICHAEL — UNIVERSITY OF MIAMI SCHOOL OF MEDICINE
- Study coordinator: BENATAR, MICHAEL
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Conditions: Amyotrophic Lateral Sclerosis Motor Neuron Disease