Ignite Creation Date:
2024-05-06 @ 2:08 PM
Last Modification Date:
2024-10-26 @ 1:25 PM
Study NCT ID:
NCT04220021
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-05-06
First Post:
2020-01-03
Brief Title:
Safety and Therapeutic Potential of the FDA-approved Drug Metformin for C9orf72 ALSFTD
Sponsor:
University of Florida
Organization:
University of Florida
Study Overview
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
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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
Detailed Description:
The C9orf72 repeat expansion is the most common cause of amyotrophic lateral sclerosis and frontotemporal dementia C9-ALSFTD 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-ALSFTD 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-ALSFTD
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| UF2019-001 | OTHER | None | None |
| OCR20620 | OTHER | None | None |
| UF2019-001 | OTHER | UF Protocol ID | None |