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Quick Info
Status
Currently Recruiting
Estimated Enrollment
18
Phase
2
Treatment Type
Single group assignment
Trial Type
open label
Primary Investigator
Laura Ranum, PhD
Contact Information
Locations
United States, Florida
UF Health at the University of Florida, Gainesville, FL, 32610, United States
Enrollment Criteria
Breathing Ability
Percent lung function (FVC) or (SVC)
N/A
Months Since Onset
Number of months since first symptoms of ALS.
N/A
Non-Invasive Ventilation (NIV)
Can PALS use a BiPAP in the trial?
N/A
Diaphragm Pacer (DPS)
Can PALS use a DPS in the trial?
N/A
Edaravone Usage
Can a PALS use edaravone (Radicut/Radicava) while enrolled in the trial?
Unknown
Update Notes
No significant updates.
5/15/2020
Location Removed UF Brain Institute, Gainesville, FL
4/1/2020
Updated recruitment status
1/14/2020
New trial added
1/7/2020

Other Information

Purpose
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.
Eligibility
Must have C9orf72 expansion repeat,
Details
The C9orf72 repeat expansion is the most common cause of amyotrophic lateral sclerosis and frontotemporal dementia (C9-ALS/FTD). Metformin, a well-tolerated diabetes drug, blocks a key pathway for expression of toxic proteins produced from the C9orf72 repeat expansion via repeat associated non-canonical start codon - in RNA (non-AUG) (RAN) translation. In mouse model of C9-ALS/FTD, metformin treatment decreases RAN protein levels and improves disease features. This current study is a small-scale clinical trial to assess the safety and potential efficacy of metformin for the treatment of C9-ALS/FTD.
Collaborator(s)
  • University of Florida
Trial Protocol as Published on Clinicaltrials.gov
NCT04220021 (First Published: 1/7/2020)