Could a common diabetes drug help fight ALS?

NCT ID NCT04220021

Ongoing Disease control Sponsor: University of Florida Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 2 times

Summary

This study tests whether metformin, a widely used diabetes drug, is safe for people with a specific genetic form of ALS (C9orf72). Over 24 weeks, 41 participants will take metformin to see if it reduces toxic proteins linked to the disease. The goal is to determine if metformin could be a viable treatment option for this type of ALS.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: Metformin (a common diabetes drug)
What this could lead to: If successful, this could point toward a new treatment option for people with a genetic form of ALS by reducing harmful proteins in the body.
What could go wrong: This is a small, early-phase trial focused on safety, not effectiveness. Metformin may not slow the disease or help all ALS patients.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

amyotrophic lateral sclerosis frontotemporal dementia frontotemporal dementia and/or amyotrophic lateral sclerosis 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

C9ORF72 AMYOTROPHIC LATERAL SCLEROSIS (ALS) FRONTOTEMPORAL DEMENTIA

Contacts and locations

Locations

UF Health at the University of Florida

Gainesville, Florida, 32610, United States

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