Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Randomized Controlled Trial (STOP PEDS RCT)
The STOP PEDS RCT is a multicenter, parallel, open label randomized controlled trial evaluating the long-term (one year) and short-term safety and efficacy of two antibiotic treatment strategies for the management of outpatient pulmonary exacerbations (PEx) in the pediatric CF population.
• Age
‣ For main cohort and non-HEMT cohort: age 6 to \<19 years
⁃ For preschool cohort: age 3 to \<6 years
• Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:
‣ sweat chloride ≥ 60 mEq/liter
⁃ two disease-causing variants in the cystic fibrosis transmembrane conductive regulator (CFTR) gene
• Written informed consent (and assent when applicable) obtained from participant or participant's legal representative and ability of participant to comply with the requirements of the study
• Highly Effective Modulator Therapy
‣ For main cohort and preschool cohort: Taking ETI or ivacaftor for at least 3 months at enrollment
⁃ For non-HEMT cohort: not eligible for HEMT based on CFTR genotype or eligible but not taking for at least 3 months and no plans to start HEMT in the next year, and also not taking tezacaftor-ivacaftor or lumacaftor-ivacaftor for at least 3 months
• For main cohort and non-HEMT cohort: able to perform acceptable and reproducible spirometry
• For main cohort and non-HEMT cohort: ppFEV1 ≥ 50% predicted at enrollment based on the Global lung Initiative (GLI) reference equations
• Ability to receive text messages and access the internet