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Streamlined Treatment of Pulmonary Exacerbations in Pediatrics (STOP-PEDS)

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ClinicalTrials.gov Identifier: NCT04608019
Recruitment Status : Recruiting
First Posted : October 29, 2020
Last Update Posted : March 25, 2021
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
University of Washington, the Collaborative Health Studies Coordinating Center

Tracking Information
First Submitted Date  ICMJE October 23, 2020
First Posted Date  ICMJE October 29, 2020
Last Update Posted Date March 25, 2021
Actual Study Start Date  ICMJE November 10, 2020
Estimated Primary Completion Date June 30, 2022   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 23, 2020)
Delayed antibiotics [ Time Frame: 28 days ]
The proportion of participants in the tailored arm who did not take any oral antibiotics in the 28 days following randomization
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 23, 2020)
  • Consent [ Time Frame: 6 months ]
    Proportion of approached patients consenting to enroll
  • Pulmonary Exacerbations Reported [ Time Frame: 18 months ]
    Proportion of Pulmonary Exacerbations in which symptoms are reported within 7 days of onset
  • Randomization Criteria [ Time Frame: 18 months ]
    Proportion of Pulmonary Exacerbation events meeting randomization criteria
  • Participant Exacerbations [ Time Frame: 18 months ]
    Proportion of enrolled participants experiencing a randomizable Pulmonary Exacerbation
  • Randomized Exacerbations [ Time Frame: 18 months ]
    Proportion of randomizable Pulmonary Exacerbations that undergo randomization
  • Day 28 Follow-up [ Time Frame: 18 months ]
    Proportion of participants with a randomized Pulmonary Exacerbation that attends an in-person Day 28 follow up visit
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
Official Title  ICMJE Streamlined Treatment of Pulmonary Exacerbations in Pediatrics (Pilot)
Brief Summary STOP PEDS is a pilot study of children with CF ages 6-18 across 10 sites in North America. The primary goal is to assess the acceptability and feasibility of a multicenter randomized trial comparing immediate antibiotics versus tailored therapy for pulmonary exacerbation (PEx) treatment in this population.
Detailed Description

STOP PEDS is a pilot study of children with CF ages 6-18 across 10 sites in North America. The primary goal is to assess the acceptability and feasibility of a multicenter randomized trial comparing immediate antibiotics versus tailored therapy for pulmonary exacerbation (PEx) treatment in this population. The primary endpoint is the proportion of participants in the tailored arm who did not take any oral antibiotics in the 28 days following randomization.

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.

Study Type  ICMJE Interventional
Study Phase  ICMJE Not Applicable
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Other: Immediate Antibiotics
    increase airway clearance/start oral antibiotics right away
  • Other: Tailored Treatment
    increase airway clearance and start oral antibiotics later if symptoms get worse or do not get better
Study Arms  ICMJE
  • Experimental: Immediate Antibiotics
    increased airway clearance plus early initiation of oral antibiotics
    Intervention: Other: Immediate Antibiotics
  • Experimental: Tailored Therapy
    increased airway clearance alone, with addition of antibiotics for worsening symptoms or failure to improve
    Intervention: Other: Tailored Treatment
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 23, 2020)
120
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 30, 2023
Estimated Primary Completion Date June 30, 2022   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Enrollment Inclusion Criteria:

  1. Age 6 to <19 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. 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

Enrollment Exclusion Criteria:

  1. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
  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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Traci Bartz, MS (206) 897-1904 bartzt@uw.edu
Contact: Erika Enright (206) 897-1922 eenright@uw.edu
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04608019
Other Study ID Numbers  ICMJE STOP-PEDS
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party University of Washington, the Collaborative Health Studies Coordinating Center
Study Sponsor  ICMJE University of Washington, the Collaborative Health Studies Coordinating Center
Collaborators  ICMJE Cystic Fibrosis Foundation
Investigators  ICMJE
Principal Investigator: Donald B. Sanders, MD Riley Children's Hospital, Indianapolis, IN
Principal Investigator: Margaret Rosenfeld, MD Seattle Children's Hospital, Seattle, WA
PRS Account University of Washington, the Collaborative Health Studies Coordinating Center
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP