PhenylketonuriasNCT06560736
Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria
A study testing Show Me FIRST coping skills intervention in people with Phenylketonurias, run by University of Missouri-Columbia at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 10 to 17 years
- Conditions the study is for
- Phenylketonurias
- What is being tested?
- Show Me FIRST coping skills intervention
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Oct 2024to 31 Aug 2026
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
University of Missouri
Columbia, Missouri, 65201
Shawn Christ, 573-884-8140, christse@missouri.edu
About this study
Official title: Development of Novel Psychological Assessment Tools and Anxiety Intervention for PKU: Intervention Trial
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.
Read the detailed description
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.
- Conditions
- Phenylketonurias
- Being tested
- Show Me FIRST coping skills intervention
- Medical subject headings
- Phenylketonurias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Show Me FIRST coping skills interventionBehavioural
Show Me FIRST is a flexible, transdiagnostic intervention organized around well-tested principles of youth psychotherapeutic change.
| Study group | Role | What they receive |
|---|---|---|
| Immediate Intervention Group | Other | Not described Receives: Behavioral: Show Me FIRST coping skills intervention |
| Wait List / Delayed Intervention Group | Other | Not described Receives: Behavioral: Show Me FIRST coping skills intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
PROMIS Anxiety & Depression scales
Patient-reported outcome (PRO) measure of anxiety \& depression symptoms
Measured: Weeks 0, 11, & 22
Test My Brain phone-based EMA cognitive test
Performance-based measure of cognitive skill \& attention
Measured: Weeks 0, 11, & 22
Secondary outcomes (10)
Behavior Rating Inventory of Executive Function (BRIEF)
Patient-reported outcome (PRO) measure of everyday symptoms of executive problems
Measured: Weeks 0, 11, & 22
Blood Phenylalanine Levels
Measured: Weeks 0, 5, 11, 16, & 22
ADHD Rating Scale - 4th version (ADHD-RS-IV)
Patient-reported outcome (PRO) measure of ADHD symptoms
Measured: Weeks 0, 11, & 22
Executive Function, Attention, and Speed Symptom Inventory (EASSI) for PKU
Patient-reported outcome (PRO) measure of cognitive symptoms
Measured: Weeks 0, 11, & 22
Rey Auditory Verbal Learning Test (Rey AVLT)
Performance-based measure of memory \& learning
Measured: Weeks 0, 11, & 22
Stop Signal Task (SST)
Performance-based measure of inhibitory ability
Measured: Weeks 0, 11, & 22
Revised Children's Anxiety and Depression Scale (RCADS)
Patient-reported outcome (PRO) measure of anxiety \& depression symptoms
Measured: Weeks 0, 11, & 22
Behavior & Feelings Survey (BFS)
Patient-reported outcome (PRO) measure of internalizing \& externalizing problems
Measured: Weeks 0, 11, & 22
Adolescent Insomnia Questionnaire (AIQ)
Patient-reported outcome (PRO) measure of sleep problems
Measured: Weeks 0, 11, & 22
Global Impression Scale
Patient-reported outcome (PRO) measure of overall symptom severity and improvement
Measured: Weeks 0, 11, & 22
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Aug 2024
- Study start1 Oct 2024
- Primary completion31 Aug 2026 (estimated)
- Study completion31 Aug 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PhenylketonuriasStudies of the same condition, open and under way.
- More trials run by University of Missouri-ColumbiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Wait-list Control Design
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Missouri-Columbia
- Sponsor type
- University, hospital or other
- Collaborators
- National PKU Alliance
- Responsible party
- Principal investigator, Shawn Christ, University of Missouri-Columbia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06560736
- Sponsor's protocol ID
- 2103266
- Status last verified
- Dec 2025
- First submitted
- 30 Apr 2024
- Last updated
- 23 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development of Novel Psychological Assessment Tools and Anxiety Intervention for PKU: Intervention Trial
- ClinicalTrials.gov ID
- NCT06560736
- Acronym / sponsor ID
- 2103266
- Conditions
- Phenylketonurias
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University of Missouri-ColumbiaUniversity, hospital or other
- Collaborators
- National PKU Alliance
- Study start
- 1 Oct 2024 (actual)
- Primary completion
- 31 Aug 2026 (estimated)
- Study completion
- 31 Aug 2026 (estimated)
- First posted
- 19 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Wait-list Control Design
| Arm | Type | Receives |
|---|---|---|
| Immediate Intervention Group | Other | Behavioral: Show Me FIRST coping skills intervention |
| Wait List / Delayed Intervention Group | Other | Behavioral: Show Me FIRST coping skills intervention |
Who may qualify
- Ages
- 10 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Show Me FIRST coping skills interventionShow Me FIRST is a flexible, transdiagnostic intervention organized around well-tested principles of youth psychotherapeutic change. | Behavioural | Immediate Intervention Group, Wait List / Delayed Intervention Group |
Primary outcome
PROMIS Anxiety & Depression scales
Time frame: Weeks 0, 11, & 22
Patient-reported outcome (PRO) measure of anxiety \& depression symptoms
Test My Brain phone-based EMA cognitive test
Time frame: Weeks 0, 11, & 22
Performance-based measure of cognitive skill \& attention
10 secondary outcomes
Behavior Rating Inventory of Executive Function (BRIEF)
Time frame: Weeks 0, 11, & 22
Blood Phenylalanine Levels
Time frame: Weeks 0, 5, 11, 16, & 22
ADHD Rating Scale - 4th version (ADHD-RS-IV)
Time frame: Weeks 0, 11, & 22
Executive Function, Attention, and Speed Symptom Inventory (EASSI) for PKU
Time frame: Weeks 0, 11, & 22
Rey Auditory Verbal Learning Test (Rey AVLT)
Time frame: Weeks 0, 11, & 22
Stop Signal Task (SST)
Time frame: Weeks 0, 11, & 22
Revised Children's Anxiety and Depression Scale (RCADS)
Time frame: Weeks 0, 11, & 22
Behavior & Feelings Survey (BFS)
Time frame: Weeks 0, 11, & 22
Adolescent Insomnia Questionnaire (AIQ)
Time frame: Weeks 0, 11, & 22
Global Impression Scale
Time frame: Weeks 0, 11, & 22
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2025.
- Individual participant dataSharing planned: No
Related trials for Phenylketonurias
RecruitingPhase 1
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
Phenylketonuria
RecruitingPhase 2
A Study of MZE782 in Adults With PKU
Phenylketonuria (PKU)
RecruitingPhase 3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phenylketonuria
Recruiting
Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
Carrier of Phenylketonuria, Healthy
RecruitingPhase 3
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phenylketonuria
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Show Me FIRST coping skills intervention in people with Phenylketonurias, run by University of Missouri-Columbia at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Shawn Christ (573-884-8140, christse@missouri.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PROMIS Anxiety & Depression scales (time frame: Weeks 0, 11, & 22). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Dec 2025.