Metformin for ALS

Recruiting in Palo Alto (17 mi)

Overseen by Laura Ranum, PhD

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 2

Waitlist Available

Sponsor: University of Florida

No Placebo Group

Prior Safety Data

Approved in 6 Jurisdictions

Trial Summary

What is the purpose of this trial?

This trial is testing if Metformin, a diabetes drug, is safe and effective for patients with C9orf72 ALS. The drug aims to block harmful proteins linked to their genetic mutation. Metformin has been used to treat type 2 diabetes for more than 60 years and is currently being investigated for its potential anticancer effects.

Research Team

Laura Ranum, PhD

Principal Investigator

University of Florida

Eligibility Criteria

This trial is for individuals with C9orf72 positive ALS/FTD who can take oral food and medication, have no severe allergies to Metformin or barium sulfate, and no implanted electrical devices or metal in their body. Pregnant women, those trying to conceive, breastfeeding mothers, people with recent cancer (except skin), liver disease, renal impairment (eGFR below 30 mL/min/1.73 m2), or on hepatotoxic drugs are excluded.

Inclusion Criteria

I am a woman who can have children and will take a pregnancy test before my swallow study exams.

I am willing to sign the consent form and HIPAA authorization.

I understand what participating in this study involves.

Exclusion Criteria

I haven't taken any experimental drugs recently.

I am not taking any medications that can harm my liver.

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

Treatment Details

Interventions

• Metformin (Biguanide)

Trial Overview The trial tests the safety and potential therapeutic effects of Metformin over a period of 24 weeks in patients with C9orf72 amyotrophic lateral sclerosis. It aims to see if Metformin can safely reduce harmful proteins produced by a specific genetic mutation in these patients.

Participant Groups

Group I: C9orf72 positive ALS Experimental Treatment 1 Intervention

Subjects with C9orf72 positive ALS will be instructed in the use of Metformin and receive the first dose of Metformin under supervision of the investigator during Visit 1, Day 2. Subjects will then continue on Metformin per the dose escalation schedule twice daily for 24 weeks.

Metformin is already approved in Canada, Japan, China, Switzerland for the following indications:

Approved in Canada as Glucophage for:

• Type 2 diabetes

Approved in Japan as Glucophage for:

• Type 2 diabetes

Approved in China as Glucophage for:

• Type 2 diabetes

Approved in Switzerland as Glucophage for:

• Type 2 diabetes

Who Is Running the Clinical Trial?

University of Florida

Lead Sponsor

Trials

1,428

Recruited

987,000+

Dr. Stephen J. Motew

University of Florida

Chief Executive Officer since 2024

MD cum laude from the University of Illinois at Chicago School of Medicine, Master's in Healthcare Administration from the University of North Carolina at Chapel Hill

Dr. Timothy E. Morey

University of Florida

Chief Medical Officer since 2023

MD and Bachelor's from the University of Florida

ALS Association

Collaborator

Trials

46

Recruited

17,500+