Prader Willi SyndromePhase 1/2NCT00742664
Behavioral Treatment of Obsessive-Compulsive Symptoms in Youth With Prader-Willi Syndrome: A Pilot Project
A Phase 1/2 study testing Cognitive-behavioral therapy in people with Prader Willi Syndrome and Obsessive-compulsive Disorder, run by University of South Florida at 1 site in United States.
Trial summary
Obsessive-compulsive (OC) symptoms are often present among youth with Prader-Willi Syndrome (PWS). They are also associated with considerable problems in the daily functioning of the child and his/her family. Read more about this study
- Who is it for?
- Ages 6 to 17 years
- Conditions the study is for
- Prader Willi Syndrome, Obsessive-compulsive Disorder
- What is being tested?
- Cognitive-behavioral therapy
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2008to Jul 2011
- Phase
- Phase 1/2
- 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 6 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- The child must have a genetically confirmed diagnosis of PWS;
- CY-BOCS Compulsion Score ≥ 8. The Compulsion Scale Score is being used as youth with PWS may have difficulty understanding or identifying the connection between obsessional thoughts and subsequent rituals. Therefore, use of the Total Score may provide an inaccurate depiction of symptoms (Storch et al., 2005);
- Stable on any psychotropic medications for 8 weeks prior to study entry;
- Between the ages of 6 and 17 years as the present measures are valid for use in this age span;
- At least one parent available to accompany the child to all sessions; and
- English speaking.
Who cannot join
- Current suicidal intent or risk;
- A positive diagnosis in the caregiver of mental retardation, psychosis, or other psychiatric conditions that would limit their ability to understand psychotherapy.
Trial locations and contacts
1 site in 1 country.
United States1 site
All Children's Hospital
St. Petersburg, Florida, 33701
About this study
Obsessive-compulsive (OC) symptoms are often present among youth with Prader-Willi Syndrome (PWS). They are also associated with considerable problems in the daily functioning of the child and his/her family. Although medication and behavioral treatments exist that target OC symptoms among youth without PWS, these treatments have not been thoroughly adapted for this population nor scientifically tested. Although medication has been helpful in addressing OC symptoms in several published case reports, the associated efficacy is modest and the potential for side effects is a realistic concern. Given that behavioral treatment for OC symptoms has superior efficacy to pharmacotherapy in youth without PWS without the accompanying risk for adverse side effects, it follows that an adapted version of behavioral therapy may hold promise in treating clinically problematic OC symptoms in youth with PWS. Thus, the purpose of the proposed grant is to develop and pilot-test a behavioral treatment for OC symptoms for use in youth with PWS. This study will allow us to develop and test a treatment protocol aimed at reducing OC symptoms that are clinically problematic and negatively impact functioning and quality of life in the child and his/her family.
Read the detailed description
Background: Obsessive-compulsive (OC) symptoms in youth with Prader-Willi Syndrome (PWS) are commonly present and associated with considerable functional impairment to the child and his/her family. Although pharmacological and behavioral treatments exist that target OC symptoms among youth without PWS, these treatments have not been systematically adapted for this population nor rigorously tested. Furthermore, although pharmacological interventions have shown promise in PWS in case reports, the efficacy is modest and the potential for side effects has been documented in youth with and without PWS. Given that behavioral treatment for OC symptoms has superior efficacy to pharmacotherapy in youth without PWS, without the accompanying risk for adverse side effects, it follows that an adapted version of this modality may hold promise in treating clinically problematic OC symptoms in youth with PWS.
Objectives: There are two primary study objectives: First, to develop a well-specified behaviorally oriented psychotherapy protocol that addresses OC symptoms, both food and non-food related, in youth with PWS. Second, to conduct a randomized wait-list controlled trial of the protocol in 12 youth with PWS.
Study Design: There will be two phases to this study: Phase I - Therapy development/manual writing and a small open trial; and Phase II - a small wait-list controlled pilot trial. Briefly, Phase I will concentrate on developing the treatment protocol through a process that draws on expert opinion coupled with focused interviews with parents. Based on this, a preliminary manual developed by the investigators will be piloted in 6 youth with PWS. Phase II will involve a preliminary test of the protocol in a sample of 12 families. This trial will focus primarily on feasibility issues (versus efficacy) in order to refine the manual, develop process measures to evaluate therapist's competence and adherence to manualized guidelines, train evaluators in assessment tasks, and develop data collection and coding systems. Six youth will be randomized to receive the treatment protocol, while 6 will participate in a wait-list of an equivalent time period. Diagnostic and symptom severity assessments will be conducted at appropriate time points (e.g., Screening, Baseline, Post-treatment) by a blinded, trained independent evaluator.
- Being tested
- Cognitive-behavioral therapy, Wait-list control
- Keywords
- Prader-Willi Syndrome, Compulsive behavior, Hoarding, Obsessive-compulsive disorder, Children, Treatment, genetically, confirmed, diagnosis
- Medical subject headings
- Prader-Willi Syndrome, Obsessive-Compulsive Disorder, Compulsive Behavior, Hoarding, Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cognitive-behavioral therapyBehavioural
The manual is informed by behavioral principles and provides a structured, replicable manner of treating OC symptoms in youth with PWS. The treatment is individual in nature, includes a strong family component (i.e., teaching parents to be their child's coach/therapist), and lasts for approximately 12 sessions, held in a twice-weekly format. In addition, the protocol includes several specific components: education about OC symptoms (i.e., etiology, behavioral function, etc.), developing a hierarchy of rituals to target, exposure and response prevention exercises, promoting adaptive familial responses to child behaviors, and relapse prevention and problem solving future situations.
Wait-list controlBehavioural
The subject will not receive active treatment during this interval.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Will receive 12 sessions of twice weekly psychotherapy targeting obsessive-compulsive symptoms. Receives: Behavioral: Cognitive-behavioral therapy |
| 2 | Placebo comparator | Not described Receives: Behavioral: Wait-list control |
What is being measured
The primary outcome is the main question the study is designed to answer.
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS; Scahill et al., 1997).
Measured: Baseline, Mid-Treatment, Post-treatment
Secondary outcomes (1)
Clinical Global Improvement (CGI; Guy, 1976).
Measured: Mid- and Post-treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2008 (estimated)
- Study startSep 2008
- Primary completionJul 2011
- Study completionJul 2011
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.
- More trials run by University of South FloridaThe sponsor's other registered studies.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 24 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of South Florida
- Sponsor type
- University, hospital or other
- Collaborators
- Johns Hopkins All Children's Hospital
- Investigators
- Eric Storch, Ph.D., Principal investigator, University of South Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00742664
- Sponsor's protocol ID
- FPWR-CBT-001
- Status last verified
- Jul 2011
- First submitted
- 26 Aug 2008
- Last updated
- 8 Aug 2011
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Aug 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Behavioral Treatment of Obsessive-Compulsive Symptoms in Youth With Prader-Willi Syndrome: A Pilot Project
- ClinicalTrials.gov ID
- NCT00742664
- Acronym / sponsor ID
- FPWR-CBT-001
- Keywords
- Prader-Willi Syndrome, Compulsive behavior, Hoarding, Obsessive-compulsive disorder, Children, Treatment, genetically, confirmed
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (estimated)
- Lead sponsor
- University of South FloridaUniversity, hospital or other
- Collaborators
- Johns Hopkins All Children's Hospital
- Study start
- Sep 2008
- Primary completion
- Jul 2011 (actual)
- Study completion
- Jul 2011 (actual)
- First posted
- 28 Aug 2008 (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.
Obsessive-compulsive (OC) symptoms are often present among youth with Prader-Willi Syndrome (PWS). They are also associated with considerable problems in the daily functioning of the child and his/her family. Although medication and behavioral treatments exist that target OC symptoms among youth without PWS, these treatments have not been thoroughly adapted for this population nor scientifically tested. Although medication has been helpful in addressing OC symptoms in several published case reports, the associated efficacy is modest and the potential for side effects is a realistic concern. Given that behavioral treatment for OC symptoms has superior efficacy to pharmacotherapy in youth without PWS without the accompanying risk for adverse side effects, it follows that an adapted version of behavioral therapy may hold promise in treating clinically problematic OC symptoms in youth with PWS. Thus, the purpose of the proposed grant is to develop and pilot-test a behavioral treatment for OC symptoms for use in youth with PWS. This study will allow us to develop and test a treatment protocol aimed at reducing OC symptoms that are clinically problematic and negatively impact functioning and quality of life in the child and his/her family.
Detailed description
Background: Obsessive-compulsive (OC) symptoms in youth with Prader-Willi Syndrome (PWS) are commonly present and associated with considerable functional impairment to the child and his/her family. Although pharmacological and behavioral treatments exist that target OC symptoms among youth without PWS, these treatments have not been systematically adapted for this population nor rigorously tested. Furthermore, although pharmacological interventions have shown promise in PWS in case reports, the efficacy is modest and the potential for side effects has been documented in youth with and without PWS. Given that behavioral treatment for OC symptoms has superior efficacy to pharmacotherapy in youth without PWS, without the accompanying risk for adverse side effects, it follows that an adapted version of this modality may hold promise in treating clinically problematic OC symptoms in youth with PWS.
Objectives: There are two primary study objectives: First, to develop a well-specified behaviorally oriented psychotherapy protocol that addresses OC symptoms, both food and non-food related, in youth with PWS. Second, to conduct a randomized wait-list controlled trial of the protocol in 12 youth with PWS.
Study Design: There will be two phases to this study: Phase I - Therapy development/manual writing and a small open trial; and Phase II - a small wait-list controlled pilot trial. Briefly, Phase I will concentrate on developing the treatment protocol through a process that draws on expert opinion coupled with focused interviews with parents. Based on this, a preliminary manual developed by the investigators will be piloted in 6 youth with PWS. Phase II will involve a preliminary test of the protocol in a sample of 12 families. This trial will focus primarily on feasibility issues (versus efficacy) in order to refine the manual, develop process measures to evaluate therapist's competence and adherence to manualized guidelines, train evaluators in assessment tasks, and develop data collection and coding systems. Six youth will be randomized to receive the treatment protocol, while 6 will participate in a wait-list of an equivalent time period. Diagnostic and symptom severity assessments will be conducted at appropriate time points (e.g., Screening, Baseline, Post-treatment) by a blinded, trained independent evaluator.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Behavioral: Cognitive-behavioral therapyWill receive 12 sessions of twice weekly psychotherapy targeting obsessive-compulsive symptoms. |
| 2 | Placebo comparator | Behavioral: Wait-list control |
Who may qualify
- Ages
- 6 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- The child must have a genetically confirmed diagnosis of PWS;
- CY-BOCS Compulsion Score ≥ 8. The Compulsion Scale Score is being used as youth with PWS may have difficulty understanding or identifying the connection between obsessional thoughts and subsequent rituals. Therefore, use of the Total Score may provide an inaccurate depiction of symptoms (Storch et al., 2005);
- Stable on any psychotropic medications for 8 weeks prior to study entry;
- Between the ages of 6 and 17 years as the present measures are valid for use in this age span;
- At least one parent available to accompany the child to all sessions; and
Key exclusion criteria (2 listed)
- Current suicidal intent or risk;
- A positive diagnosis in the caregiver of mental retardation, psychosis, or other psychiatric conditions that would limit their ability to understand psychotherapy.
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 |
|---|---|---|
| Cognitive-behavioral therapyThe manual is informed by behavioral principles and provides a structured, replicable manner of treating OC symptoms in youth with PWS. The treatment is individual in nature, includes a strong family component (i.e., teaching parents to be their child's coach/therapist), and lasts for approximately 12 sessions, held in a twice-weekly format. In addition, the protocol includes several specific components: education about OC symptoms (i.e., etiology, behavioral function, etc.), developing a hierarchy of rituals to target, exposure and response prevention exercises, promoting adaptive familial responses to child behaviors, and relapse prevention and problem solving future situations. | Behavioural | 1 |
| Wait-list controlThe subject will not receive active treatment during this interval. | Behavioural | 2 |
Primary outcome
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS; Scahill et al., 1997).
Time frame: Baseline, Mid-Treatment, Post-treatment
1 secondary outcome
Clinical Global Improvement (CGI; Guy, 1976).
Time frame: Mid- and Post-treatment
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 8 Aug 2011.
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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.
Eric Storch, Ph.D. principal investigator
University of South Florida
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Cognitive-behavioral therapy in people with Prader Willi Syndrome and Obsessive-compulsive Disorder, run by University of South Florida 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 6 inclusion and 2 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 8 Aug 2011. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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.
What does the primary outcome measure?
Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS; Scahill et al., 1997). (time frame: Baseline, Mid-Treatment, Post-treatment). It is what the study was designed to measure, not a finding.
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 8 Aug 2011.