Treatment of Self-Injurious Behavior in Individuals With Prader-Willi Syndrome
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT00065923 |
Recruitment Status :
Completed
First Posted : August 5, 2003
Last Update Posted : May 1, 2013
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Prader-Willi Syndrome Self-Injurious Behavior | Drug: Topiramate | Not Applicable |
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.
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 10 participants |
Allocation: | Randomized |
Intervention Model: | Crossover Assignment |
Masking: | Double |
Primary Purpose: | Treatment |
Official Title: | Topiramate Effects on SIB in Prader-Willi Syndrome |
Study Start Date : | July 2002 |
Actual Primary Completion Date : | December 2005 |


Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years to 66 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | Yes |
Inclusion Criteria
- PWS due to deletion of 15 q11-13 or uniparental disomy
- Actively engaging skin picking behavior
- Individual with PWS or legal guardian able to provide full informed consent. If legal guardian gives informed consent, then individual with PWS will give his/her assent.
- Acceptable methods of contraception
Exclusion Criteria
- Pregnant or breastfeeding
- Clinically significant suicidality or homicidality
- DSM-IV diagnosis of substance abuse or dependence within 6 months of study entry
- Cardiovascular, hepatic, renal, gastrointestinal, pulmonary, metabolic, endocrine, or other systemic disease which could interfere with treatment or assessment of PWS
- Treatment with any drug which might interact adversely with topiramate
- Medication or significant behavioral management change within 4 weeks of study entry
- Personal history or a first-degree family history of nephrolithiasis

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00065923
United States, Florida | |
University of Florida-Brain Institute | |
Gainesville, Florida, United States, 32609 |
Principal Investigator: | Nathan A. Shapira, MD, PhD | University of Florida |
ClinicalTrials.gov Identifier: | NCT00065923 |
Other Study ID Numbers: |
R03HD042818 ( U.S. NIH Grant/Contract ) |
First Posted: | August 5, 2003 Key Record Dates |
Last Update Posted: | May 1, 2013 |
Last Verified: | August 2010 |
Prader-Willi Syndrome Self-Injurious Behavior |
Prader-Willi Syndrome Syndrome Self-Injurious Behavior Disease Pathologic Processes Intellectual Disability Neurobehavioral Manifestations Neurologic Manifestations Nervous System Diseases Abnormalities, Multiple Congenital Abnormalities |
Chromosome Disorders Genetic Diseases, Inborn Obesity Overnutrition Nutrition Disorders Behavioral Symptoms Topiramate Anticonvulsants Hypoglycemic Agents Physiological Effects of Drugs |