Prader-Willi SyndromeNCT00065923
Treatment of Self-Injurious Behavior in Individuals With Prader-Willi Syndrome
A study testing Topiramate in people with Prader-Willi Syndrome and Self-Injurious Behavior, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 66 yearsHealthy volunteers can take part
- Conditions the study is for
- Prader-Willi Syndrome, Self-Injurious Behavior
- What is being tested?
- Topiramate
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2002to Dec 2005
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 66 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Florida-Brain Institute
Gainesville, Florida, 32609
About this study
Official title: Topiramate Effects on SIB in Prader-Willi Syndrome
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.
Read the 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 study's medical oversight physician and, if appropriate, the participant will be placed directly into the 4-month open-label extension phase.
- Conditions
- Prader-Willi Syndrome, Self-Injurious Behavior
- Being tested
- Topiramate
- Keywords
- Prader-Willi Syndrome, Self-Injurious Behavior
- Medical subject headings
- Prader-Willi Syndrome, Self-Injurious Behavior
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TopiramateDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2003 (estimated)
- Study startJul 2002
- Primary completionDec 2005
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- Sponsor type
- US National Institutes of Health
- Investigators
- Nathan A. Shapira, MD, PhD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00065923
- Sponsor's protocol ID
- R03HD042818
- Status last verified
- Aug 2010
- First submitted
- 1 Aug 2003
- Last updated
- 1 May 2013
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Topiramate Effects on SIB in Prader-Willi Syndrome
- ClinicalTrials.gov ID
- NCT00065923
- Acronym / sponsor ID
- R03HD042818
- Conditions
- Prader-Willi Syndrome, Self-Injurious Behavior
- Keywords
- Prader-Willi Syndrome, Self-Injurious Behavior
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)US National Institutes of Health
- Study start
- Jul 2002
- Primary completion
- Dec 2005 (actual)
- First posted
- 5 Aug 2003 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 study's medical oversight physician and, if appropriate, the participant will be placed directly into the 4-month open-label extension phase.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
Who may qualify
- Ages
- 18 to 66 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Topiramate | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2013.
- Primary family support organization for Prader-Willi SyndromeLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Nathan A. Shapira, MD, PhD principal investigator
University of Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Topiramate in people with Prader-Willi Syndrome and Self-Injurious Behavior, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 May 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 May 2013.