Behavioral Treatment of Obsessive-Compulsive Symptoms in Youth With Prader-Willi Syndrome: A Pilot Project
Condition: Prader Willi Syndrome; Obsessive-compulsive Disorder
MeSH terms: Prader-Willi Syndrome; Syndrome; Compulsive Personality Disorder; Obsessive-Compulsive Disorder
Study type: Interventional
Phase: Phase 1/Phase 2

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. 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.
    

Intervention name(s): Cognitive-behavioral therapy; Wait-list control
Intervention type(s): Behavioral; Behavioral
Intervention description(s): 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.; The subject will not receive active treatment during this interval.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary purpose: Treatment
Keywords: Prader-Willi Syndrome; Compulsive behavior; Hoarding; Obsessive-compulsive disorder; Children; Treatment; genetically; confirmed; diagnosis