Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
NCT ID: NCT06654752
Last Updated: 2025-11-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
NA
430 participants
INTERVENTIONAL
2024-12-01
2029-02-28
Brief Summary
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Detailed Description
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Based on the research priorities identified by CF families and clinicians and the results of the pilot study, 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 PEx in the pediatric CF population. The two treatment arms are immediate antibiotics and tailored therapy. In both arms, participants will be instructed to increase airway clearance at the onset of an eligible PEx. In the immediate antibiotics arm, they will also begin 14 days of oral antibiotics preselected by their primary CF providers, while in the tailored therapy they will only begin antibiotics if prespecified criteria for worsening symptoms or failure to improve are met.
The STOP PEDS study will enroll three cohorts. In the main cohort, children ages 6-18 on highly effective modulator therapy (HEMT) will be enrolled when well and followed for 12 months. Participants will be randomly assigned to a treatment arm and maintain that treatment assignment for all subsequent eligible PEx during their 12-month enrollment period. Two additional pilot cohorts, the preschool cohort (children ages 3 to 5 on HEMT) and the non-HEMT cohort (children ages 6-18 not eligible for HEMT), will be enrolled in parallel pilot studies. Participants will enroll when well and be followed through one randomized PEx.
Participants in the STOP PEDS RCT at selected sites will have the opportunity to enroll in optional substudies if eligible. These substudies include:
* Clinic throat swab substudy
* Home throat and nasal swab substudy
* Remote monitoring substudy (Home monitoring of lung function, vital signs, activity and sleep)
Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Immediate Antibiotics
Increased airway clearance plus early initiation of oral antibiotics
Immediate Oral Antibiotics
Increase airway clearance and start 14 days of preselected oral antibiotics right away
Tailored Therapy
Increased airway clearance alone, with addition of oral antibiotics for worsening symptoms or failure to improve
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
Interventions
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Immediate Oral Antibiotics
Increase airway clearance and start 14 days of preselected oral antibiotics right away
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
Eligibility Criteria
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Inclusion Criteria
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 ETI or ivacaftor 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
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
3 Years
18 Years
ALL
No
Sponsors
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Cystic Fibrosis Foundation
OTHER
University of Washington, the Collaborative Health Studies Coordinating Center
OTHER
Responsible Party
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Principal Investigators
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D. B. Sanders, MD, MS
Role: PRINCIPAL_INVESTIGATOR
Indiana University
Margaret Rosenfeld, MD, MPH
Role: PRINCIPAL_INVESTIGATOR
Seattle Children's Hospital
Locations
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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
Oregon Health & Science University
Portland, Oregon, United States
Children's Hospital of Philadelphia & University of Pennsylvania
Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh of UPMC & University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
University of Texas Southwestern & Children's Health
Dallas, Texas, United States
Texas Children's Hospital & Baylor College of Medicine
Houston, Texas, United States
Vermont Children's Hospital & University of Vermont Medical Center
Burlington, Vermont, United States
Virginia Commonwealth University
Richmond, Virginia, United States
Seattle Children's Hospital
Seattle, Washington, United States
University of Wisconsin
Madison, Wisconsin, United States
Children's Wisconsin & Medical College of Wisconsin
Milwaukee, Wisconsin, United States
British Columbia Children's Hospital
Vancouver, British Columbia, Canada
The Hospital for Sick Children & Toronto Canada CF Centre Pediatrics
Toronto, Ontario, Canada
Countries
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Central Contacts
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Facility Contacts
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Ashlyn Hastings
Role: primary
Elizabeth (Lisa) Ryan
Role: primary
Jared Lopez, BA
Role: primary
Tina Conti, BSRC, RRT, RRT-NPS, C-NPT
Role: primary
Lisa Ramos, CRC
Role: primary
Mary Cross
Role: primary
Joy Dangerfield
Role: primary
Mariah Flowers
Role: primary
Misty Thompson
Role: primary
Lisa Bendy
Role: backup
Mary Teresi
Role: primary
Jamelia Maynard
Role: primary
Ethan Ito
Role: primary
Dawn Kruse
Role: primary
Briana Escobar, CRC
Role: primary
Alyssa Perry
Role: primary
Jana Lomonte
Role: primary
Emily Schulte, RN, BSN
Role: primary
Barbara Johnson, RN
Role: primary
Karen McCarthy
Role: backup
Caroline Flowers, CRC
Role: primary
Sharon Kadon, RN, BSN, CPN
Role: primary
Pierce Nusbaum
Role: primary
Erin Donnelly
Role: primary
Adrienne DeRicco, RN
Role: primary
Audra Wiser
Role: primary
Lindsay Allen, CRC
Role: primary
Pavel Tuekam
Role: primary
Vanessa Marascio
Role: primary
Akilah Pierre-Louis, CRC
Role: primary
Sharon McNamara
Role: primary
Melanie Nelson, BS, RRT, CCRC
Role: primary
Laura Roth, CCRC
Role: primary
Madison Weir
Role: primary
Sheryl Hewko
Role: primary
References
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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.
Related Links
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STOP PEDS Pilot Study manuscript
Other Identifiers
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STOP PEDS RCT
Identifier Type: -
Identifier Source: org_study_id