Ignite Creation Date:
2024-05-05 @ 9:16 PM
Last Modification Date:
2024-10-26 @ 10:02 AM
Study NCT ID:
NCT00858689
Status:
COMPLETED
Last Update Posted:
2016-02-18
First Post:
2009-03-09
Brief Title:
Add-on Pilot Trial of Minocycline to Treat Fragile X Syndrome
Sponsor:
FRAXA Research Foundation
Organization:
FRAXA Research Foundation
Study Overview
Official Title:
Add-on Pilot Trial of Minocycline in Fragile X Syndrome
Status:
COMPLETED
Status Verified Date:
2016-02
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 the most common known inherited form of mental impairment developmental disability and autism Minocycline is an antibiotic that has recently been used to treat the mouse model for Fragile X and was found to reverse the structural abnormalities that are seen their brain cells The purpose of this research study is to determine if minocycline is an effective treatment for patients with fragile X syndrome FXS
Detailed Description:
Fragile X Syndrome FXS is the most common known inherited form of mental impairment and is also associated with a range of learning disabilities neurological problems such as seizures and behavioural difficulties For many individuals with FXS behavioral difficulties result in severe problems within the family and community particularly in the form of agitation temper outbursts hyperactivity and aggression These problems often require a variety of psychopharmacological and behavioural approaches Although a variety of medications can be helpful in FXS there are no targeted interventions based on molecular abnormalities that have been studied Defects in dendritic spine formation have been found in the brains of patients with Fragile X suggesting these structures may represent an anatomical and physiological basis for the cognitive deficits associated with this disorder Recent research has suggested that minocycline may have a specific benefit in the treatment of FXS Minocycline is an antibiotic that has been found to inhibit the activity of matrix metallo-proteinase-9 MMP-9 which is up-regulated in the hippocampus of FMR1 KO Fragile X Mental Retardation-1 Knockout mice and may be responsible for the immature dendritic spine profile of hippocampal neurons Minocycline has recently been used to treat the FXS KO mouse model for Fragile X and was found to rescue this abnormal phenotype by inducing the formation of mature dendritic spines in FMR1 KO hippocampal neurons both in vitro and in vivo Minocycline treated FXS KO mice also performed significantly better in the elevated maze a cognitive performance test that measures activity and anxiety
Exciting preclinical effects of minocycline with regard to the FXS disease model have led to this pilot proposal which is designed to generate preliminary data that could be used to support a larger clinical trial
The overall hypothesis is that minocycline is a specific molecular targeted treatment for FXS that will display beneficial effects on disruptive behaviour and possibly other associated features of FXS via a reduction in MMP-9 activity
Exciting preclinical effects of minocycline with regard to the FXS disease model have led to this pilot proposal which is designed to generate preliminary data that could be used to support a larger clinical trial
The overall hypothesis is that minocycline is a specific molecular targeted treatment for FXS that will display beneficial effects on disruptive behaviour and possibly other associated features of FXS via a reduction in MMP-9 activity
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None