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
sweat chloride ≥ 60 mEq/liter
Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the individual or the quality of the data
Receiving an acute course of oral or IV antibiotics at the time of enrollment or within the 14 days prior to enrollment. Individuals may be re-screened ≥21 days after completion of antibiotics if they are at their baseline state of health, per self-report
Treatment with systemic corticosteroids at enrollment or within the 14 days prior to enrollment. Individuals may be re- screened ≥21 days after completion of systemic corticosteroids if they are at their clinical baseline, per self-report
History of solid organ transplant
Parallel
Immediate Oral Antibiotics
Other interventionIncrease airway clearance and start 14 days of preselected oral antibiotics right away
Tailored Treatment: Oral Antibiotics only if Additional Treatment needed
Other interventionIncrease airway clearance and start preselected oral antibiotics later if symptoms get worse or do not get better according to prespecified criteria
One-year change in pulmonary function by spirometry-measured ppFEV1
Compare the difference in pulmonary function between arms by evaluating change in spirometry-measured percent predicted forced expiratory volume (ppFEV1). A spirometry test measures the amount of air a person can forcibly exhale after a deep breath (forced vital capacity, or FVC) and the amount of air they can exhale in one second (forced expiratory volume in one second, or FEV1). A lower measured value compared to the reference value indicates lung disease.
One-Year change in pulmonary function by LCI
Compare the difference in pulmonary function between arms by evaluating change in Lung Clearance Index (LCI). LCI increases when there is lung disease, which causes ventilation inhomogeneity. LCI is calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity \[FRC\]) required to reduce end-tidal nitrogen concentration to 1/40th of the original level.
Recovery to baseline by ppFEV1 and CRISS following a PEx
Compare recovery to baseline between arms over the 28-day period after the first pulmonary exacerbation (PEx), by spirometry-measured percent predicted forced expiratory volume (ppFEV1), and after all pulmonary exacerbations, measured by CRISS symptom scores. The Chronic Respiratory Infection Symptom Score (CRISS) ranges from 0 to 100, with higher scores indicating more symptoms.
Recovery to baseline by Lung Clearance Index (LCI)
Compare recovery to baseline between arms over the 28-day period after the first pulmonary exacerbation (PEx) in LCI, and after all exacerbations for symptom scores. The Chronic Respiratory Infection Symptom Score (CRISS) ranges from 0 to 100, with higher scores indicating more symptoms.
Safety Endpoint 1: Cumulative oral antibiotic exposure
Compare cumulative oral antibiotic exposure in each treatment arm.
Safety Endpoint 2: Number of respiratory illnesses
Compare the number of respiratory illnesses reported over the study period in each treatment arm.
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University of Washington, the Collaborative Health Studies Coordinating Center
Lead sponsor
Cystic Fibrosis Foundation
Collaborator
