An observational study in Cystic Fibrosis (CF), sponsored by State University of New York at Buffalo. Recruiting at 13 sites in United States. Open to participants aged 18 Months to 11 Years. Per ClinicalTrials.gov, last updated 2026-07-06.
Sponsored by State University of New York at Buffalo · Observational
This is a longitudinal, observational epidemiological study designed to estimate the prevalence of depression, anxiety, and behavior problems in children ages 18 months through 11 years with cystic fibrosis (CF).
Integration of mental health (MH) screening and treatment into cystic fibrosis (CF) care represents over 10 years of research and clinical progress, driven by elevated rates of depression and anxiety in the International Depression Epidemiological Study, MH guidelines, and CF Foundation implementation support to screen adolescents and adults in all CF Centers. Benefits of screening include earlier identification, greater access to care, reduced stigma, and positive uptake from the CF community.
However, TIDES did not include children with CF under 12 years. Depression and anxiety have increased dramatically in young children, with new guidelines for MH screening of children in primary care. Given the pediatric MH crisis and the widespread adoption of cystic fibrosis fibrosis transmembrane conductance regulator (CFTR) modulator therapy, which have been associated with adverse events, there is an urgent need to gather MH data in children with CF \<12 years. Thus, the goals of this study are to evaluate the national, longitudinal prevalence of depression, anxiety, and behavior problems in children with CF 18 months through 11 years, evaluate and compare the performance of two widely used brief screeners (criterion validity, sensitivity, specificity) to identify the optimal measures for this population, and characterize neuropsychiatric adverse events (AEs) associated with CFTR modulator therapy in this age group. Purposive randomized sampling will be used to recruit 600 children (half 18 mos.-5 yrs. and half 6-11 yrs.) at 16 CF Centers across the US. This study will estimate the prevalence of children above the clinical cut-score on each symptom domain (depression, anxiety, behavior problems) and evaluate their longitudinal course and predictors. Rigorous mixed methods will be used to describe any potential AEs perceived by parents or children to be associated with CFTR modulator therapy. This study will provide the groundwork to extend mental health screening and care to younger children with CF.
1,581 studies on the registry are indexed under Cystic Fibrosis; 190 are open to participants now.
This study's planned enrollment of 600 is above the median of 85 across 482 observational studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →State University of New York at Buffalo is the lead sponsor of 287 studies on the registry; 65 are open to participants now.
Of its 19 completed or terminated interventional studies of FDA-regulated products, 15 (79%) have results posted.
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Children with Cystic Fibrosis between 18 months and 11 years
Exclusion Criteria:
300 children with CF and their parents will be recruited. This cohort will help identify early signs of internalizing and externalizing behaviors, attention-related concerns, and the impact of CF treatments on psychosocial health. Children in this cohort may also be identified for participation in Aim 3 if they are on or eligible for modulator therapy.
300 children with CF and their parents will be recruited. Includes school-aged children who can self-report their emotional and behavioral health, beginning at age 8 years. This group will be assessed for emerging mental health symptoms, cognitive development, and procedural anxiety related to CF treatments. Children in this cohort may also be identified for participation in Aim 3 if they are on or eligible for modulator therapy.
Prevalence of depression, anxiety, and behavioral problems in children with cystic fibrosis
Estimate the national prevalence of depression, anxiety, and behavioral problems among children with cystic fibrosis aged 18 months through 11 years using the Behavior Assessment System for Children, Third Edition (BASC-3). At the baseline assessment (Time 1), parents complete the age-appropriate BASC-3 Parent Rating Scale, and children aged 8-11 years complete the BASC-3 Self-Report of Personality. BASC-3 classifications of emotional and behavioral symptoms will be used to estimate the prevalence of clinically significant symptoms.
Time frame: 07/01/2024 - 06/30/2028
Diagnostic performance of brief behavioral and mental health screening instruments
Evaluate the diagnostic accuracy and clinical utility of the Pediatric Symptom Checklist (PSC-Preschool, PSC-17, and PSC-Y-17) and PROMIS short forms for anxiety, depressive symptoms, anger/irritability, sleep disturbance, cognitive functioning, flexibility, and persistence by comparing their performance with BASC-3 at baseline.
Time frame: 07/01/2024 - 06/30/2028
Longitudinal changes in child mental health symptoms
Assess changes in parent-reported and child self-reported mental health symptoms over three study assessments using PSC and PROMIS measures. BASC-3 is administered only at the first assessment.
Time frame: 07/01/2024 - 06/30/2028
Cystic fibrosis-specific health-related quality of life
Assess CF-specific health-related quality of life using age-appropriate versions of the Cystic Fibrosis Questionnaire-Revised (CFQ-R). Parent-proxy and child self-report versions are administered according to participant age, with the Preschool Pictorial CFQ-R administered once for eligible children.
Time frame: 07/01/2024 - 06/30/2028
Parent symptoms of depression and anxiety
Assess parent mental health using the Patient Health Questionnaire-8 (PHQ-8) and Generalized Anxiety Disorder-7 (GAD-7) at each assessment.
Time frame: 07/01/2024 - 06/30/2028
Neuropsychiatric symptoms associated with CFTR modulator therapy
Evaluate parent-reported new or worsening neuropsychiatric symptoms following initiation of elexacaftor/tezacaftor/ivacaftor (ETI) or vanzacaftor/tezacaftor/deutivacaftor (VTD) using the CFTR Modulator Survey. Symptoms include anxiety, depression, attention problems, behavioral dysregulation, irritability, and sleep disturbance. The Modified Naranjo Scale will be used to assess the likelihood that reported symptoms are related to CFTR modulator therapy.
Time frame: 07/01/2024 - 06/30/2028
Qualitative experiences related to CFTR modulator-associated neuropsychiatric symptoms
Conduct semi-structured qualitative interviews with a purposive sample of parents reporting worsening neuropsychiatric symptoms following CFTR modulator initiation or non-initiation because of mental health concerns. Eligible school-aged children may also participate. Interview transcripts will undergo thematic analysis.
Time frame: 07/01/2024 - 06/30/2028
Plan to share: Yes — An application process will be established for external data requests. Initially, data access will be granted upon request by Brown University Health (BUH), with full public access only after a defined period following study completion. Beyond academic publications, BUH will contribute to the creation of publicly available study summaries, ensuring that key findings are accessible to the CF community, healthcare providers, and patient advocacy groups.
Supporting information: Study protocol, Sap, Icf, Csr, Analytic code
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State University of New York at Buffalo