Prader-Willi Syndrome
Conditions
Keywords
Prader-Willi Syndrome, Emotion dysregulation, aggression, anxiety, depression, hyperphagia
Brief summary
The goal of this study is to help teens with Prader-Willi Syndrome (PWS) and their families learn practical strategies for managing issues like irritability, meltdowns, and anxiety. The main objective of the study is: To adapt current Regulating Together materials to create an outpatient group program for emotion dysregulation in Prader-Willi Syndrome (PWS) that will improve psychosocial outcomes for youth with PWS.
Detailed description
Up to ten children with Prader-Willi Syndrome (PWS) ages 13 to 17.5 years and their caregivers will be enrolled in the study. This study is being done to develop and test a behavior therapy to help teenagers with PWS calm their emotions. Participants will complete assessments at five time points: Screening (T1); Week 0: Baseline visit (T2); Week 5: Post Active Treatment (T3); Week 10: 5 week follow-up (T4); and Week 15: 10 week follow-up (T5). Participants will receive 5 weeks of intervention, twice per week, following the T2 visit.
Interventions
BEHAVIORALRegulating Together group therapy
Regulating Together is the original curriculum developed for autistic youth that uses evidence-based therapeutic strategies including relaxation techniques, parent management training, and mindfulness to help regulate emotions. This study will adapt the original Regulating Together curriculum to focus on behavioral interventions to treat emotion dysregulation in Prader-Willi Syndrome.
Sponsors
Children's Mercy Hospital Kansas City
Foundation for Prader-Willi Research
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
13 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
Child Participant Inclusion Criteria: * Ages 13-17.5 years * Confirmed genetic diagnosis of Prader-Willi Syndrome * Fluent in spoken English * Adolescent's use of flexible phrase speech or greater * Meeting clinically significant emotional dysregulation criteria * Willing to participate in weekly 90-minute group sessions * Family is willing to keep prescribed psychiatric medication and outside behavioral interventions stable * Parent, guardian, or legally authorized representative must provide written permission on behalf of the participant Child Participant
Exclusion criteria
* Initiation of new psychosocial intervention within 30 days prior to first day of treatment * Presence of physical aggression in the adolescent directed towards a peer outside the home that resulted in injury within 30 days prior to screening * Presence of comorbid major neuropsychiatric illness warranting other treatment approaches * Presence of any major sensory impairment that would limit participating in the material including blindness or uncorrected hearing loss Caregiver Inclusion Criteria: * Age ≥ 18 years * Lives and cares for their child with PWS for \> 50% of the year * Fluent in spoken English * Willing to participate in twice weekly 90-minute sessions, including one virtual session weekly Caregiver
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Emotion Dysregulation Inventory - Reactivity | From enrollment to the end of their follow up appointment, about 10 weeks after therapy has completed | The Emotion Dysregulation Inventory consists of two subscales. We will use the Reactivity subscale which captures poorly regulated negative emotional responses and a dysphoria subscale characterized by increased negative affect and lack of motivation. Minimum raw score: 0; Maximum raw score: 96; Higher scores reflect higher emotional reactivity. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Clinician Global Impressions - Improvement (CGI-I) | From enrollment to the end of their follow up appointment, about 10 weeks after therapy has completed | A trained, independent clinician will rate the CGI at T3 and T5. The CGI-I provides a qualitative measure of treatment response through a rating from 1=very much improved to 7=very much worse. |
| Parenting Stress Index, 4th Edition, Short Form | From enrollment to the end of their follow up appointment, about 10 weeks after therapy has completed | A caregiver-report questionnaire measuring the magnitude of stress in the parent-child system. Minimum score: 36; Maximum score: 180; Higher scores reflect greater parental stress |
| Aberrant Behavior Checklist, 2nd Edition (ABC-2) | From enrollment to the end of their follow up appointment, about 10 weeks after therapy has completed | Irritability subscale; a caregiver-report measures to measure change in irritability and related challenging behaviors. Minimum score: 0; Maximum score: 45; higher scores reflect greater irritability |
| Caregiver and Adolescent Readiness and Satisfaction Surveys | From enrollment to end of follow up at 15 weeks | We will assess acceptability as reflected in the average caregiver and adolescent satisfaction rating measured using two study-specific measures. Caregivers will complete the Caregiver Readiness and Satisfaction Survey (CRS) and adolescents will complete the Adolescent Readiness and Satisfaction Survey (ARS). |
| Emotion Regulation Skills Test | From enrollment to the end of their follow up appointment, about 10 weeks after therapy has completed | A child self-report measure created for Regulating Together to measure knowledge of skills taught to manage emotion dysregulation. |
| Patient-Reported Outcomes Measurement Information System (PROMIS) Anxiety and Depression Scales (Parent-Proxy) | From enrollment to the end of their follow up appointment, about 10 weeks after therapy has completed | Caregiver-report measures to measure change in internalizing symptoms. For both measures: minimum raw value: 13; maximum raw value: 65; higher scores reflect greater anxiety/depression |
Countries
United States
Contacts
CONTACTGrayson Manley, RN
PRINCIPAL_INVESTIGATORWalker McKinney, PhD
Children's Mercy
Outcome results
None listed