Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALS/FTD
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|ClinicalTrials.gov Identifier: NCT04220021|
Recruitment Status : Recruiting
First Posted : January 7, 2020
Last Update Posted : September 16, 2022
|Condition or disease||Intervention/treatment||Phase|
|C9orf72 Amyotrophic Lateral Sclerosis (ALS) Frontotemporal Dementia||Drug: Metformin||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||58 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Single-Center, Open Label Study to Assess the Safety and Tolerability of Metformin in Subjects With C9orf72 Amyotrophic Lateral Sclerosis Over 24 Weeks of Treatment|
|Actual Study Start Date :||January 10, 2020|
|Estimated Primary Completion Date :||April 6, 2023|
|Estimated Study Completion Date :||April 6, 2023|
Experimental: C9orf72 positive ALS
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 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.
Other Name: Metformin hydrochloride sustained-release (SR)
- Number of subjects with treatment-emergent adverse events [Safety and Tolerability] [ Time Frame: Baseline through 24 weeks ]The safety and tolerability of Metformin in participants with C9orf72 ALS currently treated with Metformin will be evaluated by the number of subjects with treatment-emergent adverse events
- Change in RAN protein levels [ Time Frame: baseline through week 24 ]Assess the RAN protein levels in cerebrospinal fluid (CSF) samples from participants at specific intervals.
- Change in ALS Functional Rating Scale (ALSFRS-R) score [ Time Frame: Baseline through Week 52 ]The ALSFRS-R is a quickly administered (5 minute) ordinal rating scale (ratings 0-4) used to determine subjects' assessment of their capability and independence in 12 functional activities/questions. Scores of 4 equaling 'normal' and scores of 0 equaling total lack of ability.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04220021
|Contact: Deborah Morrison, MAfirstname.lastname@example.org|
|United States, Florida|
|UF Health at the University of Florida||Recruiting|
|Gainesville, Florida, United States, 32610|
|Contact: Deborah Morrison, MA 352-273-5189 email@example.com|
|Principal Investigator: Laura Ranum, PHD|
|Sub-Investigator: James Wymer, MD, PHD|
|Sub-Investigator: Emily Plowman, PHD, CCC-SLP|
|Principal Investigator:||Laura Ranum, PhD||University of Florida|