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RecruitingPhenylketonurias

Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria

Eligible age

10–17 yrs

Accepts

All genders

Locations

1 state

Healthy volunteers

No

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About this study

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.

Sponsor: University of Missouri-Columbia

You may qualify if…

  • 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.

You may not qualify if…

  • 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

Where it's recruiting

Missouri

Columbia

Source: ClinicalTrials.gov · NCT06560736 · last updated 2025-12-23