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

Primary Purpose

Cystic Fibrosis

Status

Completed

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

Immediate Antibiotics

Tailored Treatment

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring cystic fibrosis, oral antibiotics, pulmonary exacerbation, pediatric

Eligibility Criteria

6 Years - 18 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Enrollment Inclusion Criteria:

Age 6 to <19 years
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
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
Able to perform acceptable and reproducible spirometry
FEV1 ≥ 50% predicted at enrollment based on the Global lung Initiative (GLI) reference equations
At least 1 course of oral or IV antibiotics for respiratory symptoms since January 1, 2019.
Ability to receive text messages and access the internet

Enrollment Exclusion Criteria:

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.
Previous randomization in the study
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.
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.
History of solid organ transplant
History of positive culture for Mycobacterium abscessus in the 12 months prior to enrollment
Treatment with antibiotics for any non-tuberculous mycobacteria (NTM) at enrollment
Three or more IV antibiotic-treated PEx in the 12 months prior to enrollment
Treatment with chronic oral antibiotics other than azithromycin at enrollment

Sites / Locations

Tucson Cystic Fibrosis Center
Children's Hospital of Colorado
Children's Healthcare of Atlanta
Lurie Children's Hospital of Chicago & Northwestern University
Riley Hospital for Children
Helen DeVos Children's Hospital
Oregon Health Sciences University
Children's Hospital of Pittsburgh of UPMC
Texas Children's Hospital and Baylor College of Medicine
Seattle Children's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Immediate Antibiotics

Tailored Therapy

Arm Description

increased airway clearance plus early initiation of oral antibiotics

increased airway clearance alone, with addition of antibiotics for worsening symptoms or failure to improve

Outcomes

Primary Outcome Measures

Delayed antibiotics

The proportion of participants in the tailored arm who did not take any oral antibiotics in the 28 days following randomization

Secondary Outcome Measures

Consent

Proportion of approached patients consenting to enroll

Pulmonary Exacerbations Reported

Proportion of Pulmonary Exacerbations in which symptoms are reported within 7 days of onset

Randomization Criteria

Proportion of Pulmonary Exacerbation events meeting randomization criteria

Participant Exacerbations

Proportion of enrolled participants experiencing a randomizable Pulmonary Exacerbation

Randomized Exacerbations

Proportion of randomizable Pulmonary Exacerbations that undergo randomization

Day 28 Follow-up

Proportion of participants with a randomized Pulmonary Exacerbation that attends an in-person Day 28 follow up visit

Full Information

NCT ID

NCT04608019

First Posted

October 23, 2020

Last Updated

July 20, 2023

Sponsor

University of Washington, the Collaborative Health Studies Coordinating Center

Collaborators

Cystic Fibrosis Foundation

1. Study Identification

Unique Protocol Identification Number

NCT04608019

Brief Title

Streamlined Treatment of Pulmonary Exacerbations in Pediatrics

Acronym

STOP-PEDS

Official Title

Streamlined Treatment of Pulmonary Exacerbations in Pediatrics (Pilot)

Study Type

Interventional

2. Study Status

Record Verification Date

July 2023

Overall Recruitment Status

Completed

Study Start Date

November 10, 2020 (Actual)

Primary Completion Date

August 18, 2022 (Actual)

Study Completion Date

August 18, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

University of Washington, the Collaborative Health Studies Coordinating Center

Collaborators

Cystic Fibrosis Foundation

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis

Keywords

cystic fibrosis, oral antibiotics, pulmonary exacerbation, pediatric

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

121 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Immediate Antibiotics

Arm Type

Experimental

Arm Description

increased airway clearance plus early initiation of oral antibiotics

Arm Title

Tailored Therapy

Arm Type

Experimental

Arm Description

increased airway clearance alone, with addition of antibiotics for worsening symptoms or failure to improve

Intervention Type

Other

Intervention Name(s)

Immediate Antibiotics

Intervention Description

increase airway clearance/start oral antibiotics right away

Intervention Type

Other

Intervention Name(s)

Tailored Treatment

Intervention Description

increase airway clearance and start oral antibiotics later if symptoms get worse or do not get better

Primary Outcome Measure Information:

Title

Delayed antibiotics

Description

The proportion of participants in the tailored arm who did not take any oral antibiotics in the 28 days following randomization

Time Frame

28 days

Secondary Outcome Measure Information:

Title

Consent

Description

Proportion of approached patients consenting to enroll

Time Frame

6 months

Title

Pulmonary Exacerbations Reported

Description

Proportion of Pulmonary Exacerbations in which symptoms are reported within 7 days of onset

Time Frame

18 months

Title

Randomization Criteria

Description

Proportion of Pulmonary Exacerbation events meeting randomization criteria

Time Frame

18 months

Title

Participant Exacerbations

Description

Proportion of enrolled participants experiencing a randomizable Pulmonary Exacerbation

Time Frame

18 months

Title

Randomized Exacerbations

Description

Proportion of randomizable Pulmonary Exacerbations that undergo randomization

Time Frame

18 months

Title

Day 28 Follow-up

Description

Proportion of participants with a randomized Pulmonary Exacerbation that attends an in-person Day 28 follow up visit

Time Frame

18 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Enrollment Inclusion Criteria: Age 6 to <19 years 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: sweat chloride ≥ 60 mEq/liter two disease-causing variants in the cystic fibrosis transmembrane conductive regulator (CFTR) gene 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 Able to perform acceptable and reproducible spirometry FEV1 ≥ 50% predicted at enrollment based on the Global lung Initiative (GLI) reference equations At least 1 course of oral or IV antibiotics for respiratory symptoms since January 1, 2019. Ability to receive text messages and access the internet Enrollment Exclusion Criteria: 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. Previous randomization in the study 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. 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. History of solid organ transplant History of positive culture for Mycobacterium abscessus in the 12 months prior to enrollment Treatment with antibiotics for any non-tuberculous mycobacteria (NTM) at enrollment Three or more IV antibiotic-treated PEx in the 12 months prior to enrollment Treatment with chronic oral antibiotics other than azithromycin at enrollment

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Donald B. Sanders, MD

Organizational Affiliation

Riley Children's Hospital, Indianapolis, IN

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Margaret Rosenfeld, MD

Organizational Affiliation

Seattle Children's Hospital, Seattle, WA

Official's Role

Principal Investigator

Facility Information:

Facility Name

Tucson Cystic Fibrosis Center

City

Tucson

State/Province

Arizona

ZIP/Postal Code

85713

Country

United States

Facility Name

Children's Hospital of Colorado

City

Aurora

State/Province

Colorado

ZIP/Postal Code

80045

Country

United States

Facility Name

Children's Healthcare of Atlanta

City

Atlanta

State/Province

Georgia

ZIP/Postal Code

30322

Country

United States

Facility Name

Lurie Children's Hospital of Chicago & Northwestern University

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60611-2605

Country

United States

Facility Name

Riley Hospital for Children

City

Indianapolis

State/Province

Indiana

ZIP/Postal Code

46202

Country

United States

Facility Name

Helen DeVos Children's Hospital

City

Grand Rapids

State/Province

Michigan

ZIP/Postal Code

49503

Country

United States

Facility Name

Oregon Health Sciences University

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239

Country

United States

Facility Name

Children's Hospital of Pittsburgh of UPMC

City

Pittsburgh

State/Province

Pennsylvania

ZIP/Postal Code

15224

Country

United States

Facility Name

Texas Children's Hospital and Baylor College of Medicine

City

Houston

State/Province

Texas

ZIP/Postal Code

77030

Country

United States

Facility Name

Seattle Children's Hospital

City

Seattle

State/Province

Washington

ZIP/Postal Code

98105

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Citations:

PubMed Identifier

11401068

Citation

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.

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