Ignite Creation Date:
2024-05-05 @ 11:31 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00065923
Status:
COMPLETED
Last Update Posted:
2013-05-01
First Post:
2003-08-01
Brief Title:
Treatment of Self-Injurious Behavior in Individuals With Prader-Willi Syndrome
Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Organization:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Study Overview
Official Title:
Topiramate Effects on SIB in Prader-Willi Syndrome
Status:
COMPLETED
Status Verified Date:
2010-08
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:
Prader-Willi syndrome PWS is a genetic disorder usually caused by the deletion of a specific gene One of the symptoms of PWS is self-injurious behavior SIB a common form of SIB in PWS patients is skin picking The injury may be severe enough to require frequent medical attention This trial will evaluate SIB in individuals with PWS and will test the effectiveness of the drug topiramate to control SIB
Detailed Description:
PWS is a neurogenetic disorder resulting from a loss of the paternal-only expressed genes on chromosome 15 15 q11-13 PWS is characterized by a persistent pattern of SIB most notably skin picking that results in frequent medical care and attention SIB in mental retardation and related developmental disabilities is often monitored by behavioral observation methods Direct evaluation of skin lesions has been reported to help systematically follow wounds and wound healing However there are differences between the type and body location of SIB in individuals with PWS as compared to those with mental retardation The goal of this study is to characterize SIB in PWS and to evaluate the efficacy of topiramate versus placebo in attenuating SIB in individuals with PWS
A preliminary 8-week open-label study conducted to evaluate topiramate for appetite and weight in PWS has shown good tolerability and beneficial effects of topiramate During that study an unexpected and serendipitous finding was that of the six participants four engaged in SIB and all four had noticeable symptom improvement during the 8 weeks of treatment Three of these four have continued on topiramate therapy long term with positive results in terms of decreased self-injury
Participants in the study will be randomized to receive either topiramate or a placebo for 6 weeks All participants will be monitored for SIB by observation and photographic recordings of the resultant skin lesions by reports of group home staff and by standardized rating measurements of self-injury At the end of 6 weeks participants receiving topiramate will receive decreasing doses of topiramate participants receiving placebo will continue to receive the placebo At week 9 participants previously receiving topiramate will be given placebo and participants previously receiving placebo will be given topiramate After 6 weeks all participants will be entered into a 4-month open-label extension phase Safety and efficacy measurements will be assessed during the 15 study visits in the event of worsening SIB the blind will be broken by the studys medical oversight physician and if appropriate the participant will be placed directly into the 4-month open-label extension phase
A preliminary 8-week open-label study conducted to evaluate topiramate for appetite and weight in PWS has shown good tolerability and beneficial effects of topiramate During that study an unexpected and serendipitous finding was that of the six participants four engaged in SIB and all four had noticeable symptom improvement during the 8 weeks of treatment Three of these four have continued on topiramate therapy long term with positive results in terms of decreased self-injury
Participants in the study will be randomized to receive either topiramate or a placebo for 6 weeks All participants will be monitored for SIB by observation and photographic recordings of the resultant skin lesions by reports of group home staff and by standardized rating measurements of self-injury At the end of 6 weeks participants receiving topiramate will receive decreasing doses of topiramate participants receiving placebo will continue to receive the placebo At week 9 participants previously receiving topiramate will be given placebo and participants previously receiving placebo will be given topiramate After 6 weeks all participants will be entered into a 4-month open-label extension phase Safety and efficacy measurements will be assessed during the 15 study visits in the event of worsening SIB the blind will be broken by the studys medical oversight physician and if appropriate the participant will be placed directly into the 4-month open-label extension phase
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