Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
STOP PEDS RCT
1 other identifier
interventional
430
2 countries
33
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for not_applicable
Started Dec 2024
Longer than P75 for not_applicable
33 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 14, 2024
CompletedFirst Posted
Study publicly available on registry
October 23, 2024
CompletedStudy Start
First participant enrolled
December 1, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 30, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
February 28, 2029
March 10, 2026
March 1, 2026
3.1 years
October 14, 2024
March 6, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
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.
1 year
Secondary Outcomes (1)
One-Year change in pulmonary function by LCI
1 year
Other Outcomes (51)
Recovery to baseline by ppFEV1 and CRISS following a PEx
28 days
Recovery to baseline by Lung Clearance Index (LCI)
28 days
Safety Endpoint 1: Cumulative oral antibiotic exposure
1 year
- +48 more other outcomes
Study Arms (2)
Immediate Antibiotics
EXPERIMENTALIncreased airway clearance plus early initiation of oral antibiotics
Tailored Therapy
EXPERIMENTALIncreased airway clearance alone, with addition of oral antibiotics for worsening symptoms or failure to improve
Interventions
Increase airway clearance and start 14 days of preselected oral antibiotics right away
Increase airway clearance and start preselected oral antibiotics later if symptoms get worse or do not get better according to prespecified criteria
Eligibility Criteria
You may qualify if:
- 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 HEMT 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
You may not qualify if:
- 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
- History of positive culture for Mycobacterium abscessus in the 12 months prior to enrollment
- Treatment with antibiotics for any non-tuberculous mycobacteria (NTM) at enrollment
- Three or more IV antibiotic-treated PEx in the 12 months prior to enrollment
- Treatment with chronic oral antibiotics other than azithromycin at enrollment
- Treatment with systemic corticosteroids for allergic bronchopulmonary aspergillosis (ABPA) in the 12 months prior to enrollment
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (33)
The Children's Hospital Alabama & University of Alabama at Birmingham
Birmingham, Alabama, 35233, United States
Tucson Cystic Fibrosis Center
Tucson, Arizona, 85713, United States
Children's Hospital of Los Angeles & Anton Yelchin Cystic Fibrosis Clinic
Los Angeles, California, 90027, United States
Stanford University
Palo Alto, California, 94304, United States
Rady Children's Hospital at University of California San Diego
San Diego, California, 92123, United States
Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
Children's Healthcare of Atlanta & Emory University
Atlanta, Georgia, 30322, United States
Ann & Robert H. Lurie Children's Hospital of Chicago & Northwestern University
Chicago, Illinois, 60611, United States
Riley Hospital for Children & Indiana University
Indianapolis, Indiana, 46202, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Johns Hopkins Hospital, Johns Hopkins University
Baltimore, Maryland, 21287, United States
Boston Children's Hospital & Harvard University
Boston, Massachusetts, 02115, United States
University of Michigan Health System
Ann Arbor, Michigan, 48109-5212, United States
Children's Hospitals and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
The Minnesota Cystic Fibrosis Center & University of Minnesota
Minneapolis, Minnesota, 55455, United States
Children's Mercy Hospital
Kansas City, Missouri, 64108, United States
St. Louis Children's Hospital & Washington University School of Medicine
St Louis, Missouri, 63110, United States
University of Rochester Medical Center Strong Memorial
Rochester, New York, 14642, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Oregon Health & Science University
Portland, Oregon, 97239-3098, United States
Children's Hospital of Philadelphia & University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Children's Hospital of Pittsburgh of UPMC & University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
University of Texas Southwestern & Children's Health
Dallas, Texas, 75235, United States
Texas Children's Hospital & Baylor College of Medicine
Houston, Texas, 77030, United States
Vermont Children's Hospital & University of Vermont Medical Center
Burlington, Vermont, 05401, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
Seattle Children's Hospital
Seattle, Washington, 98105, United States
University of Wisconsin
Madison, Wisconsin, 53792, United States
Children's Wisconsin & Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4, Canada
The Hospital for Sick Children & Toronto Canada CF Centre Pediatrics
Toronto, Ontario, M5G 0A4, Canada
Related Publications (1)
Sanders DB, Bartz TM, Zemanick ET, Hoppe JE, Hinckley Stukovsky KD, Cogen JD, Bendy L, McNamara S, Enright E, Kime NA, Kronmal RA, Edwards TC, Morgan WJ, Rosenfeld M. A Pilot Randomized Clinical Trial of Pediatric Cystic Fibrosis Pulmonary Exacerbations Treatment Strategies. Ann Am Thorac Soc. 2023 Dec;20(12):1769-1776. doi: 10.1513/AnnalsATS.202303-245OC.
PMID: 37683122RESULT
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
D. B. Sanders, MD, MS
Indiana University
- PRINCIPAL INVESTIGATOR
Margaret Rosenfeld, MD, MPH
Seattle Children's Hospital
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 14, 2024
First Posted
October 23, 2024
Study Start
December 1, 2024
Primary Completion (Estimated)
December 30, 2027
Study Completion (Estimated)
February 28, 2029
Last Updated
March 10, 2026
Record last verified: 2026-03
Data Sharing
- IPD Sharing
- Will not share