Treatment options for pulmonary exacerbations in children with cystic fibrosis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

The STOP PEDS RCT is a multicenter, open-label randomized controlled trial that evaluates the safety and efficacy of two antibiotic treatment strategies for managing outpatient pulmonary exacerbations in pediatric patients with cystic fibrosis. Participants aged 3 to 18 will be assigned to either an immediate antibiotics group or a tailored therapy group, where antibiotics are only administered if specific worsening symptoms occur. The study aims to determine the long-term and short-term outcomes of these treatment approaches over a 12-month period, focusing on reducing unnecessary antibiotic exposure while effectively managing exacerbations.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Age

   1. For main cohort and non-HEMT cohort: age 6 to \<19 years
   2. For preschool cohort: age 3 to \<6 years
2. 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:

   1. sweat chloride ≥ 60 mEq/liter
   2. two disease-causing variants in the cystic fibrosis transmembrane conductive regulator (CFTR) gene
3. 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
4. Highly Effective Modulator Therapy

   1. For main cohort and preschool cohort: Taking HEMT for at least 3 months at enrollment
   2. 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
5. For main cohort and non-HEMT cohort: able to perform acceptable and reproducible spirometry
6. For main cohort and non-HEMT cohort: ppFEV1 ≥ 50% predicted at enrollment based on the Global lung Initiative (GLI) reference equations
7. Ability to receive text messages and access the internet

Exclusion Criteria:

1. 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
2. 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
3. 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
4. History of solid organ transplant
5. History of positive culture for Mycobacterium abscessus in the 12 months prior to enrollment
6. Treatment with antibiotics for any non-tuberculous mycobacteria (NTM) at enrollment
7. Three or more IV antibiotic-treated PEx in the 12 months prior to enrollment
8. Treatment with chronic oral antibiotics other than azithromycin at enrollment
9. Treatment with systemic corticosteroids for allergic bronchopulmonary aspergillosis (ABPA) in the 12 months prior to enrollment

Where it's running

• The Children's Hospital Alabama & University of Alabama at Birmingham
  Birmingham, Alabama, United States Recruiting
• Tucson Cystic Fibrosis Center
  Tucson, Arizona, United States Recruiting
• Children's Hospital of Los Angeles & Anton Yelchin Cystic Fibrosis Clinic
  Los Angeles, California, United States Recruiting
• Stanford University
  Palo Alto, California, United States Recruiting
• Children's Hospital of Colorado
  Aurora, Colorado, United States Recruiting
• Children's Healthcare of Atlanta & Emory University
  Atlanta, Georgia, United States Recruiting

Study contacts

• Principal investigator: D. B. Sanders, MD, MS — Indiana University
• Study coordinator: Erika Enright
• Email: eenright@uw.edu
• Phone: 206-897-1922

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.