RECRUITINGINTERVENTIONAL
Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Randomized Controlled Trial (STOP PEDS RCT)
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
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 willing to sign a consent form (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
Who Should NOT Join This Trial:
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
...See full criteria on ClinicalTrials.gov
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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
Treatments Being Tested
OTHER
Immediate Oral Antibiotics
Increase airway clearance and start 14 days of preselected oral antibiotics right away
OTHER
Tailored Treatment: Oral Antibiotics only if Additional Treatment needed
Increase airway clearance and start preselected oral antibiotics later if symptoms get worse or do not get better according to prespecified criteria
Locations (20)
The Children's Hospital Alabama & University of Alabama at Birmingham
Birmingham, Alabama, United States
Tucson Cystic Fibrosis Center
Tucson, Arizona, United States
Children's Hospital of Los Angeles & Anton Yelchin Cystic Fibrosis Clinic
Los Angeles, California, United States
Stanford University
Palo Alto, California, United States
Rady Children's Hospital at University of California San Diego
San Diego, California, United States
Children's Hospital of Colorado
Aurora, Colorado, United States
Children's Healthcare of Atlanta & Emory University
Atlanta, Georgia, United States
Ann & Robert H. Lurie Children's Hospital of Chicago & Northwestern University
Chicago, Illinois, United States
Riley Hospital for Children & Indiana University
Indianapolis, Indiana, United States
University of Iowa
Iowa City, Iowa, United States
Johns Hopkins Hospital, Johns Hopkins University
Baltimore, Maryland, United States
Boston Children's Hospital & Harvard University
Boston, Massachusetts, United States
University of Michigan Health System
Ann Arbor, Michigan, United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, United States
The Minnesota Cystic Fibrosis Center & University of Minnesota
Minneapolis, Minnesota, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
St. Louis Children's Hospital & Washington University School of Medicine
St Louis, Missouri, United States
University of Rochester Medical Center Strong Memorial
Rochester, New York, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States