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NCT Number: NCT06560736

Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria

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.

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Key information

Age range

10 year–17 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Not applicable

Primary location

University of Missouri

Columbia, Missouri, 65201, United States

Location status: Recruiting

Location contact

Shawn Christ

CONTACT

[email protected]

573-884-8140

About this study

A sample of 30 adolescents (age 10-17) with PKU and a history of elevated anxiety and/or depression symptomatology will be recruited to participate in a randomized control trial. Following screening and informed consent, participants will be randomized to an immediate intervention group (n=15) or wait-list/delayed intervention group (n=15). Participants will receive the 10-week Show Me First program for treatment of anxiety and depression. Participants will complete extensive psychological and neurocognitive evaluations at three timepoints: Weeks 0, 11, and 22. The assessments and intervention will all be administered remotely via secure videoconference and cell-phone based applications.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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.

Exclusion criteria

  • 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

Treatment and study plan

Show Me FIRST coping skills intervention

Behavioral

Show Me FIRST is a flexible, transdiagnostic intervention organized around well-tested principles of youth psychotherapeutic change.

Primary outcomes

  1. PROMIS Anxiety & Depression scales

    Time frame: Weeks 0, 11, & 22

    Patient-reported outcome (PRO) measure of anxiety & depression symptoms

  2. Test My Brain phone-based EMA cognitive test

    Time frame: Weeks 0, 11, & 22

    Performance-based measure of cognitive skill & attention

Secondary outcomes

  1. Behavior Rating Inventory of Executive Function (BRIEF)

    Time frame: Weeks 0, 11, & 22

    Patient-reported outcome (PRO) measure of everyday symptoms of executive problems

  2. Blood Phenylalanine Levels

    Time frame: Weeks 0, 5, 11, 16, & 22

  3. ADHD Rating Scale - 4th version (ADHD-RS-IV)

    Time frame: Weeks 0, 11, & 22

    Patient-reported outcome (PRO) measure of ADHD symptoms

  4. Executive Function, Attention, and Speed Symptom Inventory (EASSI) for PKU

    Time frame: Weeks 0, 11, & 22

    Patient-reported outcome (PRO) measure of cognitive symptoms

  5. Rey Auditory Verbal Learning Test (Rey AVLT)

    Time frame: Weeks 0, 11, & 22

    Performance-based measure of memory & learning

  6. Stop Signal Task (SST)

    Time frame: Weeks 0, 11, & 22

    Performance-based measure of inhibitory ability

  7. Revised Children's Anxiety and Depression Scale (RCADS)

    Time frame: Weeks 0, 11, & 22

    Patient-reported outcome (PRO) measure of anxiety & depression symptoms

  8. Behavior & Feelings Survey (BFS)

    Time frame: Weeks 0, 11, & 22

    Patient-reported outcome (PRO) measure of internalizing & externalizing problems

  9. Adolescent Insomnia Questionnaire (AIQ)

    Time frame: Weeks 0, 11, & 22

    Patient-reported outcome (PRO) measure of sleep problems

  10. Global Impression Scale

    Time frame: Weeks 0, 11, & 22

    Patient-reported outcome (PRO) measure of overall symptom severity and improvement

Study contacts

Contact information is provided by the study sponsor or research team.

Shawn Christ

CONTACT

[email protected]

573-884-8140

Sponsors and collaborators

Lead sponsor

University of Missouri-Columbia

Other

Collaborators

  • National PKU Alliance

Registry information

Official study title

Development of Novel Psychological Assessment Tools and Anxiety Intervention for PKU: Intervention Trial

Important dates

Study start
2024
Primary completion
2026
Study completion
2026
First posted
Aug 19, 2024
Registry last updated
Dec 23, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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