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Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD

NCT04220021 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 41

Last updated 2025-12-02

Study results available
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Summary

The primary objective is to assess the safety and tolerability of Metformin in subjects with C9orf72 amyotrophic lateral sclerosis administered for 24 weeks. The overall objective is to determine if Metformin is safe in C9orf72 ALS patients and is a potentially viable therapeutic treatment for C9-ALS that reduces repeat-associated non-canonical start codon - in DNA (non-ATG) (RAN) proteins that are produced by the C9orf72 repeat expansion mutation.

Conditions

  • C9orf72 Amyotrophic Lateral Sclerosis (ALS)
  • Frontotemporal Dementia

Interventions

DRUG

Metformin

Metformin is a widely used, well-tolerated drug that has been used for decades as a first-line defense for treating type 2 diabetes. Its safety has been well established. Subjects will begin treatment with Metformin at a dosage of 500mg with an escalation of dosage by 500mg every week to a maximal dosage of 2000mg. Dosing will be twice daily.

Sponsors & Collaborators

  • ALS Association

    collaborator OTHER

  • United States Department of Defense

    collaborator FED

  • University of Florida

    lead OTHER

Principal Investigators

  • Laura Ranum, PhD · University of Florida

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Max Age
80 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-01-10
Primary Completion
2024-08-26
Completion
2026-06-30
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04220021 on ClinicalTrials.gov