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

Streamlined Treatment of Pulmonary Exacerbations in Pediatrics (Pilot)

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.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Other: Immediate Antibiotics; Other: Tailored Treatment

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: Interventional
• Enrollment (Actual): 121
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Tucson, Arizona, United States, 85713
      • Tucson Cystic Fibrosis Center
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital of Colorado
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Children's Healthcare of Atlanta
  • Illinois
    • Chicago, Illinois, United States, 60611-2605
      • Lurie Children's Hospital of Chicago & Northwestern University
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Riley Hospital for Children
  • Michigan
    • Grand Rapids, Michigan, United States, 49503
      • Helen DeVos Children's Hospital
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health Sciences University
  • Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15224
      • Children's Hospital of Pittsburgh of UPMC
  • Texas
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital and Baylor College of Medicine
  • Washington
    • Seattle, Washington, United States, 98105
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Immediate Antibiotics; increased airway clearance plus early initiation of oral antibiotics	Other: Immediate Antibiotics; increase airway clearance/start oral antibiotics right away
Experimental: Tailored Therapy; increased airway clearance alone, with addition of antibiotics for worsening symptoms or failure to improve	Other: Tailored Treatment; increase airway clearance and start oral antibiotics later if symptoms get worse or do not get better

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Delayed antibiotics	The proportion of participants in the tailored arm who did not take any oral antibiotics in the 28 days following randomization	28 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Consent	Proportion of approached patients consenting to enroll	6 months
Pulmonary Exacerbations Reported	Proportion of Pulmonary Exacerbations in which symptoms are reported within 7 days of onset	18 months
Randomization Criteria	Proportion of Pulmonary Exacerbation events meeting randomization criteria	18 months
Participant Exacerbations	Proportion of enrolled participants experiencing a randomizable Pulmonary Exacerbation	18 months
Randomized Exacerbations	Proportion of randomizable Pulmonary Exacerbations that undergo randomization	18 months
Day 28 Follow-up	Proportion of participants with a randomized Pulmonary Exacerbation that attends an in-person Day 28 follow up visit	18 months

Collaborators and Investigators

• Sponsor: University of Washington, the Collaborative Health Studies Coordinating Center
• Collaborators: Cystic Fibrosis Foundation
• Investigators: Principal Investigator: Donald B. Sanders, MD, Riley Children's Hospital, Indianapolis, IN; Principal Investigator: Margaret Rosenfeld, MD, Seattle Children's Hospital, Seattle, WA

Publications and helpful links

General 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. 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. Erratum In: Ann Am Thorac Soc. 2024 Jul;21(7):1113. doi: 10.1513/AnnalsATS.21i7Erratum.

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): November 10, 2020
• Primary Completion (Actual): August 18, 2022
• Study Completion (Actual): August 18, 2022

Study Registration Dates

• First Submitted: October 23, 2020
• First Submitted That Met QC Criteria: October 23, 2020
• First Posted (Actual): October 29, 2020

Study Record Updates

• Last Update Posted (Actual): October 18, 2024
• Last Update Submitted That Met QC Criteria: October 16, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Keywords: pediatric; oral antibiotics; cystic fibrosis; pulmonary exacerbation
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis; Anti-Infective Agents; Anti-Bacterial Agents
• Other Study ID Numbers: STOP-PEDS

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No