Could a common diabetes drug help fight ALS?
NCT ID NCT04220021
First seen Jun 25, 2026 · Last updated Aug 21, 2026 · Updated 4 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.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
41 people
The number who actually took part.
- Started
-
Jan 2020
- Expected to finish
-
Aug 2027
An estimate. End dates often move.
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 to 80 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Subjects have a diagnosis of probable or definite ALS in accordance with the Revisited El-Escorial Criteria. * Subjects have a likely diagnosis of chromosome 9 open reading frame 72 (C9orf72) positive ALS/FTD. * Subjects must be currently on an oral diet and able to take foods, pills and liquids by mouth equivalent to a score of 4 or above on the Functional Oral Intake Scale * Subjects must have no known allergy to barium sulfate or Metformin. * Subjects or subject's legally authorized representative must be willing and able to complete informed consent/assent and HIPAA authorization. * Ability to comprehend and be informed of the nature of the study, as assessed by the PI or Co-Investigators. * Subjects prescribed to take Metformin at or before the time of first dosing. (The study is open to subjects currently taking Metformin or subjects who have taken Metformin in the past). * Availability to participate for the entire study duration. * Female subjects of childbearing potential must have a negative urine pregnancy test prior to Videofluoroscopic Swallow Study (VFSS) exam during Visit 1, 3, and 4. Exclusion Criteria: * Subjects who score 3 or below on the Functional Oral Intake Scale * Subjects who do not carry the C9ORF72 hexanucleotide repeat expansion as determined by laboratory analysis. * Subjects with a history of clinically significant liver disease, renal disease, or any other medical condition judged to be exclusionary by the investigator. * Subjects who are unwilling to sign informed consent or subjects who for any other reason in the judgment of investigator are unable to complete the study. * Female subjects who have a positive urine pregnancy test (βhCG) at screening or visit 1, are trying to become pregnant or are breastfeeding. * Subjects with active cancer within the previous 2 years, except treated basal cell carcinoma of the skin. * Subjects who have taken any experimental drug within 30 days prior to enrollment or within 5 half-lives of the investigational drug -whichever is the longer period. * Subjects with known history or presence of moderate or severe renal impairment as defined by an estimated glomerular filtration rate (eGFR) value below 30 mL/min/1.73 m2. * Subjects with hepatic impairment as defined by baseline elevations of serum aminotransferases greater than 5 times upper limit of normal or evidence of liver dysfunction (e.g., elevated bilirubin). * Use of potentially hepatotoxic drugs: (e.g., allopurinol, methyldopa, sulfasalazine). * Subjects with clinically significant abnormal laboratory values in the judgment of the investigator. * Subject with implanted electrical device (i.e. cardiac pacemaker or a neurostimulator), metal or metallic clip(s) in their body (i.e. an aneurysm clip in the brain) that will be damaged by participation in the MRI portion of the study. * Anything else that, in the opinion of the investigator, would place the subject at increased risk or preclude the subject's full compliance with or completion of the study.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for C9ORF72 amyotrophic lateral sclerosis (ALS) are added.
Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
UF Health at the University of Florida
Gainesville, Florida, 32610, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a massive data registry crack the code of Parkinson's and related brain diseases?
- Spinal injection drug targets genetic cause of ALS and dementia
- Can a nudge in your chart unlock Life-Saving genetic clues?
- Could a brain disease change behavior years before diagnosis?
- Can a synthetic cannabinoid calm agitation in frontotemporal dementia?
- Can sound waves soothe Dementia's emotional turmoil?