Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Pilot Study
NCT ID: NCT04608019
Last Updated: 2024-10-18
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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COMPLETED
NA
121 participants
INTERVENTIONAL
2020-11-10
2022-08-18
Brief Summary
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Detailed Description
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Ultimately, we want to learn:
* What is the best way to treat pulmonary exacerbations?
* Should everyone with a pulmonary exacerbation take antibiotics?
* Do the benefits of starting antibiotics at the first signs of illness outweigh the possible risks, like side effects and antibiotic resistance?
This pilot study is designed to determine if an interventional study to help answer these questions is feasible. Up to 120 participants will be enrolled and followed through their well state of health, then for 28 days following their first randomized exacerbation. Enrollment will stop after 80 pulmonary exacerbation events have been randomized, even if this does not require 120 participants. Due to the nature of the study, the identity of treatment assignment will be known to investigators, research staff, and patients (ie, not blinded).
Total duration of this pilot study is expected to be approximately 18 months: 6 months for participant recruitment and 12 months for follow up. Participants could be monitored for up to 18 months if they do not have an exacerbation. However, it is anticipated that the majority of participants will experience a randomizable PEx event and therefore have a shorter follow up period.
Conditions
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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 Antibiotics
increase airway clearance/start oral antibiotics right away
Tailored Therapy
increased airway clearance alone, with addition of antibiotics for worsening symptoms or failure to improve
Tailored Treatment
increase airway clearance and start oral antibiotics later if symptoms get worse or do not get better
Interventions
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Immediate Antibiotics
increase airway clearance/start oral antibiotics right away
Tailored Treatment
increase airway clearance and start oral antibiotics later if symptoms get worse or do not get better
Eligibility Criteria
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Inclusion Criteria
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. Able to perform acceptable and reproducible spirometry
5. FEV1 ≥ 50% predicted at enrollment based on the Global lung Initiative (GLI) reference equations
6. At least 1 course of oral or IV antibiotics for respiratory symptoms since January 1, 2019.
7. Ability to receive text messages and access the internet
Exclusion Criteria
2. Previous randomization in the study
3. Receiving antibiotics for a PEx at the time of enrollment or within the 21 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.
4. Treatment with systemic corticosteroids at enrollment. Individuals may be re- screened ≥21 days after completion of systemic corticosteroids if they are at their clinical baseline, per self-report.
5. History of solid organ transplant
6. History of positive culture for Mycobacterium abscessus in the 12 months prior to enrollment
7. Treatment with antibiotics for any non-tuberculous mycobacteria (NTM) at enrollment
8. Three or more IV antibiotic-treated PEx in the 12 months prior to enrollment
9. Treatment with chronic oral antibiotics other than azithromycin at enrollment
6 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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Donald B. Sanders, MD
Role: PRINCIPAL_INVESTIGATOR
Riley Children's Hospital, Indianapolis, IN
Margaret Rosenfeld, MD
Role: PRINCIPAL_INVESTIGATOR
Seattle Children's Hospital, Seattle, WA
Locations
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Tucson Cystic Fibrosis Center
Tucson, Arizona, United States
Children's Hospital of Colorado
Aurora, Colorado, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Lurie Children's Hospital of Chicago & Northwestern University
Chicago, Illinois, United States
Riley Hospital for Children
Indianapolis, Indiana, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, United States
Oregon Health Sciences University
Portland, Oregon, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
Texas Children's Hospital and Baylor College of Medicine
Houston, Texas, United States
Seattle Children's Hospital
Seattle, Washington, United States
Countries
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References
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Bradley J, McAlister O, Elborn S. Pulmonary function, inflammation, exercise capacity and quality of life in cystic fibrosis. Eur Respir J. 2001 Apr;17(4):712-5. doi: 10.1183/09031936.01.17407120.
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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Related Info
Other Identifiers
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STOP-PEDS
Identifier Type: -
Identifier Source: org_study_id
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