Ignite Creation Date:
2024-05-06 @ 12:18 PM
Last Modification Date:
2024-10-26 @ 12:56 PM
Study NCT ID:
NCT03722290
Status:
COMPLETED
Last Update Posted:
2021-02-04
First Post:
2018-10-24
Brief Title:
Metformin in Children and Adults With Fragile X Syndrome
Sponsor:
Université de Sherbrooke
Organization:
Université de Sherbrooke
Study Overview
Official Title:
Evaluate the Efficacy and Safety of Metformin in Children and Adults With Fragile X Syndrome an Open-label Study
Status:
COMPLETED
Status Verified Date:
2021-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Fragile X Syndrome FXS is caused by loss of FMR1 expression on the X chromosome that leads to increased mRNA translation which results in hyperactivation of ERK extracellular signal-regulated kinase and mTORC1 mechanistic target of rifampicin complex 1 signalling and consequently in synaptic dysfunction and neurological development There is presently no cure for FXS Recent studies suggest that metformin a widely prescribed drug for type II diabetes in children and adults which crosses the blood-brain barrier corrects various neurological and behavioral FXS phenotypes by normalizing ERK signaling EIF4E phosphorylation and lowering expression of MMP9 to normal Since this drug has not been previously used specifically for treatment of FXS only few cases reported the investigators propose an open-label trial of metformin in children and adults with FXS to better understand the safety and efficacy in both behavior and cognition
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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