While previous PKU intervention research has largely focused on pharmacological treatment of elevated Phe levels, the adaptation of evidence-based psychosocial therapy holds promise for addressing cognitive and psychological symptoms and significantly improving quality of life. Study goals include: (1) to demonstrate the efficacy and feasibility of a short-term skills-based intervention (Show Me FIRST) for anxiety and depression in adolescents with PKU, and (2) to further establish the psychometric validity of novel assessment measures recently developed by the Investigators that utilize ecological momentary assessment (EMA) to capture "in the moment" neurocognitive and psychological function. The Investigators propose to conduct a randomized controlled trial wherein 30 adolescents with PKU will be randomized to either an immediate intervention group or wait-list/delayed intervention group.
Youth 10-17 years old and at least one parent/guardian
Formal diagnosis of PKU (as confirmed/reported by caregiver): identified by newborn screening with PKU as evidenced by a blood Phe level ≥ 360μmol/L; received treatment within the first 30 days of life.
Interest in receiving a brief, psychotherapeutic skills program to prevent and treat symptoms of anxiety and/or depression, according to parent- and youth-report
Located in Missouri, Kansas, or Illinois.
Have significant intellectual disability (IQ<70) or significant expressive or receptive language that would interfere with psychotherapy, according to parent- or youth-report
Plans to receive non-study psychotherapy during the course of study participation, according to parent- or youth-report
Plans to change current psychoactive or PKU-related medication during the course of study participation, according to parent- or youth-report
Have active suicidal ideation that would necessitate immediate treatment or require higher level of care than brief psychotherapy, according to parent- or youth-report
Parallel
Show Me FIRST coping skills intervention
BehavioralShow Me FIRST is a flexible, transdiagnostic intervention organized around well-tested principles of youth psychotherapeutic change.
PROMIS Anxiety & Depression scales
Patient-reported outcome (PRO) measure of anxiety \& depression symptoms
Test My Brain phone-based EMA cognitive test
Performance-based measure of cognitive skill \& attention
Behavior Rating Inventory of Executive Function (BRIEF)
Patient-reported outcome (PRO) measure of everyday symptoms of executive problems
Blood Phenylalanine Levels
ADHD Rating Scale - 4th version (ADHD-RS-IV)
Patient-reported outcome (PRO) measure of ADHD symptoms
Executive Function, Attention, and Speed Symptom Inventory (EASSI) for PKU
Patient-reported outcome (PRO) measure of cognitive symptoms
Click on the lead sponsor to view all of their trials.
University of Missouri-Columbia
Lead sponsor
National PKU Alliance
Collaborator
