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Sponsored by: |
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
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Information provided by: | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
ClinicalTrials.gov Identifier: | NCT00065923 |
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.
Condition | Intervention |
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Prader-Willi Syndrome Self-Injurious Behavior |
Drug: Topiramate |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Double-Blind, Placebo Control, Crossover Assignment, Safety/Efficacy Study |
Official Title: | Topiramate Effects on SIB in Prader-Willi Syndrome |
Estimated Enrollment: | 10 |
Study Start Date: | July 2002 |
Estimated Study Completion Date: | June 2005 |
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 study’s medical oversight physician and, if appropriate, the participant will be placed directly into the 4-month open-label extension phase.
Ages Eligible for Study: | 18 Years to 66 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | Yes |
Inclusion Criteria
Exclusion Criteria
Contact: Nathan A. Shapira, MD, PhD | 352-392-3681 | shapira@psychiatry.ufl.edu |
United States, Florida | |
University of Florida-Brain Institute | Recruiting |
Gainesville, Florida, United States, 32609 | |
Contact: Nikki Ricciuti, RN 352-265-0162 nikkir@psychiatry.ufl.edu | |
Principal Investigator: Nathan A. Shapira, MD, PhD | |
Sub-Investigator: Daniel J. Driscoll, PhD, MD |
Principal Investigator: | Nathan A. Shapira, MD, PhD | University of Florida, McKnight Brain Institute |
Study ID Numbers: | R03HD42818 |
Study First Received: | August 1, 2003 |
Last Updated: | June 23, 2005 |
ClinicalTrials.gov Identifier: | NCT00065923 |
Health Authority: | United States: Federal Government |
Prader-Willi Syndrome Self-Injurious Behavior |
Chromosomal abnormalities Obesity Chromosome Disorders Prader-Willi syndrome Behavioral Symptoms Mental Retardation Genetic Diseases, Inborn Abnormalities, Multiple |
Topiramate Neurologic Manifestations Nutrition Disorders Overnutrition Prader-Willi Syndrome Congenital Abnormalities Neurobehavioral Manifestations Self-Injurious Behavior |
Anti-Obesity Agents Pathologic Processes Disease Therapeutic Uses Syndrome Physiological Effects of Drugs |
Nervous System Diseases Protective Agents Neuroprotective Agents Central Nervous System Agents Pharmacologic Actions Anticonvulsants |