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

• ClinicalTrials.gov Identifier: NCT04608019
• Recruitment Status: Active, not recruiting
• First Posted: October 29, 2020
• Last Update Posted: December 23, 2021
• Sponsor: University of Washington, the Collaborative Health Studies Coordinating Center
• Collaborator: Cystic Fibrosis Foundation
• Information provided by (Responsible Party): University of Washington, the Collaborative Health Studies Coordinating Center

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Cystic Fibrosis	Other: Immediate Antibiotics; Other: Tailored Treatment	Not Applicable

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 121 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Streamlined Treatment of Pulmonary Exacerbations in Pediatrics (Pilot)
• Actual Study Start Date: November 10, 2020
• Estimated Primary Completion Date: June 30, 2022
• Estimated Study Completion Date: June 30, 2023

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. 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

Secondary Outcome Measures :

1. Consent [ Time Frame: 6 months ]
   Proportion of approached patients consenting to enroll
2. Pulmonary Exacerbations Reported [ Time Frame: 18 months ]
   Proportion of Pulmonary Exacerbations in which symptoms are reported within 7 days of onset
3. Randomization Criteria [ Time Frame: 18 months ]
   Proportion of Pulmonary Exacerbation events meeting randomization criteria
4. Participant Exacerbations [ Time Frame: 18 months ]
   Proportion of enrolled participants experiencing a randomizable Pulmonary Exacerbation
5. Randomized Exacerbations [ Time Frame: 18 months ]
   Proportion of randomizable Pulmonary Exacerbations that undergo randomization
6. 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

Eligibility Criteria

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

Criteria

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

Contacts and Locations

Locations

United States, Arizona

Tucson Cystic Fibrosis Center

Tucson, Arizona, United States, 85713

United States, Colorado

Children's Hospital of Colorado

Aurora, Colorado, United States, 80045

United States, Georgia

Children's Healthcare of Atlanta

Atlanta, Georgia, United States, 30322

United States, Illinois

Lurie Children's Hospital of Chicago & Northwestern University

Chicago, Illinois, United States, 60611-2605

United States, Indiana

Riley Hospital for Children

Indianapolis, Indiana, United States, 46202

United States, Michigan

Helen DeVos Children's Hospital

Grand Rapids, Michigan, United States, 49503

United States, Oregon

Oregon Health Sciences University

Portland, Oregon, United States, 97239

United States, Pennsylvania

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States, 15224

United States, Texas

Texas Children's Hospital and Baylor College of Medicine

Houston, Texas, United States, 77030

United States, Washington

Seattle Children's Hospital

Seattle, Washington, United States, 98105

Sponsors and Collaborators

University of Washington, the Collaborative Health Studies Coordinating Center

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

More Information

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.

• Responsible Party: University of Washington, the Collaborative Health Studies Coordinating Center
• ClinicalTrials.gov Identifier: NCT04608019
• Other Study ID Numbers: STOP-PEDS
• First Posted: October 29, 2020
• Last Update Posted: December 23, 2021
• Last Verified: December 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by University of Washington, the Collaborative Health Studies Coordinating Center:

cystic fibrosis; oral antibiotics; pulmonary exacerbation; pediatric

Additional relevant MeSH terms:

Cystic Fibrosis; Fibrosis; Pathologic Processes; Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Anti-Bacterial Agents; Anti-Infective Agents