ClinicalTrials.gov record NCT06560736
Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria
Development of Novel Psychological Assessment Tools and Anxiety Intervention for PKU: Intervention Trial
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This randomized study tests a 10-week remote skills-based program called Show Me FIRST for adolescents with PKU who have elevated anxiety or depression symptoms. Thirty participants are randomized to immediate treatment or a wait-list/delayed treatment group, with assessments at baseline, week 11, and week 22. The study also uses phone-based ecological momentary assessment (EMA) tools to capture real-time cognitive and emotional function.
What it is studying. The study tests a short, skills-based psychotherapy program delivered remotely to address anxiety and depression in adolescents with PKU. It also evaluates the reliability and validity of novel phone-based ecological momentary assessment (EMA) measures of cognition and mood.
What the researchers hope to learn. Scientific goals: to test the feasibility and effects of the 10-week Show Me FIRST program on anxiety and depression measures in adolescents with PKU; to establish psychometric validity of novel EMA-based cognitive and mood measures; and to collect data on executive function, attention, memory, ADHD symptoms, sleep, global impression, and blood phenylalanine over time.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Behavioral
- Show Me FIRST coping skills intervention
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
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
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
Participants are randomized to start a 10-week remote skills program (Show Me FIRST) either immediately or after a wait-list delay. All sessions and study contacts are delivered remotely by secure videoconference and phone-based apps. Participants complete psychological and neurocognitive assessments at Weeks 0, 11, and 22, perform phone-based ecological momentary cognitive tests, and provide blood samples for phenylalanine levels at Weeks 0, 5, 11, 16, and 22.
How the study is structured. Randomized, parallel-group interventional trial with no masking. Participants are assigned to either immediate 10-week Show Me FIRST treatment or a wait-list/delayed treatment group. Therapy and assessments are delivered remotely by videoconference and phone apps. Psychological and cognitive evaluations (including repeated EMA) and blood phenylalanine measurements are collected at planned timepoints.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on phenylketonuria
Important dates
- Study start
- Oct 1, 2024
- Primary completion
- Aug 31, 2026 (estimated)
- Study completion
- Aug 31, 2026 (estimated)
- Target enrollment
- 30 participants (estimated)
- Last updated on registry
- Dec 23, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- University of Missouri-Columbia
- Principal investigator
- Shawn Christ, Associate Professor of Psychological Sciences
University of Missouri-Columbia - Collaborators
- National PKU Alliance
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Shawn Christ 📞 573-884-8140 ✉ christse@missouri.edu
Participating sites (1)
- University of MissouriRecruiting
Columbia, Missouri, 65201 · United States
- Shawn Christ · 573-884-8140 · christse@missouri.edu
Terms used on this page · explanations by MedicalUpdates.ai
- randomized
- Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- Exclusion Criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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