RECRUITING

Streamlined Treatment of Pulmonary Exacerbations in Pediatrics

Conditions

Cystic Fibrosis oral antibiotics pulmonary exacerbation pediatric

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The STOP PEDS RCT is a multicenter, parallel, open label randomized controlled trial evaluating the long-term (one year) and short-term safety and efficacy of two antibiotic treatment strategies for the management of outpatient pulmonary exacerbations (PEx) in the pediatric CF population.

Official Title

Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Randomized Controlled Trial (STOP PEDS RCT)

Quick Facts

Study Start:2024-12-01

Study Completion:2029-02-28

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06654752

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:3 Years to 18 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion Criteria
1. Age 1. For main cohort and non-HEMT cohort: age 6 to \<19 years 2. For preschool cohort: age 3 to \<6 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. Highly Effective Modulator Therapy 1. For main cohort and preschool cohort: Taking ETI or ivacaftor for at least 3 months at enrollment 2. For non-HEMT cohort: not eligible for HEMT based on CFTR genotype or eligible but not taking for at least 3 months and no plans to start HEMT in the next year, and also not taking tezacaftor-ivacaftor or lumacaftor-ivacaftor for at least 3 months 5. For main cohort and non-HEMT cohort: able to perform acceptable and reproducible spirometry 6. For main cohort and non-HEMT cohort: ppFEV1 ≥ 50% predicted at enrollment based on the Global lung Initiative (GLI) reference equations 7. Ability to receive text messages and access the internet	1. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the individual or the quality of the data 2. Receiving an acute course of oral or IV antibiotics at the time of enrollment or within the 14 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 3. Treatment with systemic corticosteroids at enrollment or within the 14 days prior to enrollment. Individuals may be re- screened ≥21 days after completion of systemic corticosteroids if they are at their clinical baseline, per self-report 4. History of solid organ transplant 5. History of positive culture for Mycobacterium abscessus in the 12 months prior to enrollment 6. Treatment with antibiotics for any non-tuberculous mycobacteria (NTM) at enrollment 7. Three or more IV antibiotic-treated PEx in the 12 months prior to enrollment 8. Treatment with chronic oral antibiotics other than azithromycin at enrollment 9. Treatment with systemic corticosteroids for allergic bronchopulmonary aspergillosis (ABPA) in the 12 months prior to enrollment

Inclusion Criteria

Exclusion Criteria

1. Age
1. For main cohort and non-HEMT cohort: age 6 to \<19 years
2. For preschool cohort: age 3 to \<6 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. Highly Effective Modulator Therapy
1. For main cohort and preschool cohort: Taking ETI or ivacaftor for at least 3 months at enrollment
2. For non-HEMT cohort: not eligible for HEMT based on CFTR genotype or eligible but not taking for at least 3 months and no plans to start HEMT in the next year, and also not taking tezacaftor-ivacaftor or lumacaftor-ivacaftor for at least 3 months
5. For main cohort and non-HEMT cohort: able to perform acceptable and reproducible spirometry
6. For main cohort and non-HEMT cohort: ppFEV1 ≥ 50% predicted at enrollment based on the Global lung Initiative (GLI) reference equations
7. Ability to receive text messages and access the internet

1. Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the individual or the quality of the data
2. Receiving an acute course of oral or IV antibiotics at the time of enrollment or within the 14 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
3. Treatment with systemic corticosteroids at enrollment or within the 14 days prior to enrollment. Individuals may be re- screened ≥21 days after completion of systemic corticosteroids if they are at their clinical baseline, per self-report
4. History of solid organ transplant
5. History of positive culture for Mycobacterium abscessus in the 12 months prior to enrollment
6. Treatment with antibiotics for any non-tuberculous mycobacteria (NTM) at enrollment
7. Three or more IV antibiotic-treated PEx in the 12 months prior to enrollment
8. Treatment with chronic oral antibiotics other than azithromycin at enrollment
9. Treatment with systemic corticosteroids for allergic bronchopulmonary aspergillosis (ABPA) in the 12 months prior to enrollment

Contacts and Locations

Study Contact

Erika Enright

CONTACT

206-897-1922

eenright@uw.edu

Principal Investigator

D. B. Sanders, MD, MS

PRINCIPAL_INVESTIGATOR

Indiana University

Margaret Rosenfeld, MD, MPH

PRINCIPAL_INVESTIGATOR

Seattle Children's Research Institute

Study Locations (Sites)

The Children's Hospital Alabama & University of Alabama at Birmingham

Birmingham, Alabama, 35233

United States

Tucson Cystic Fibrosis Center

Tucson, Arizona, 85713

United States

Children's Hospital of Los Angeles & Anton Yelchin Cystic Fibrosis Clinic

Los Angeles, California, 90027

United States

Stanford University

Palo Alto, California, 94304

United States

Rady Children's Hospital at University of California San Diego

San Diego, California, 92123

United States

Children's Hospital of Colorado

Aurora, Colorado, 80045

United States

Children's Healthcare of Atlanta & Emory University

Atlanta, Georgia, 30322

United States

Ann & Robert H. Lurie Children's Hospital of Chicago & Northwestern University

Chicago, Illinois, 60611

United States

Riley Hospital for Children & Indiana University

Indianapolis, Indiana, 46202

United States

University of Iowa

Iowa City, Iowa, 52242

United States

Johns Hopkins Hospital, Johns Hopkins University

Baltimore, Maryland, 21287

United States

Boston Children's Hospital & Harvard University

Boston, Massachusetts, 02115

United States

University of Michigan Health System

Ann Arbor, Michigan, 48109-5212

United States

Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, 55404

United States

The Minnesota Cystic Fibrosis Center & University of Minnesota

Minneapolis, Minnesota, 55455

United States

Children's Mercy Hospital

Kansas City, Missouri, 64108

United States

St. Louis Children's Hospital & Washington University School of Medicine

Saint Louis, Missouri, 63110

United States

University of Rochester Medical Center Strong Memorial

Rochester, New York, 14642

United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27514

United States

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229

United States

Oregon Health & Science University

Portland, Oregon, 97239-3098

United States

Children's Hospital of Philadelphia & University of Pennsylvania

Philadelphia, Pennsylvania, 19104

United States

Children's Hospital of Pittsburgh of UPMC & University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15224

United States

Medical University of South Carolina

Charleston, South Carolina, 29425

United States

University of Texas Southwestern & Children's Health

Dallas, Texas, 75235

United States

Texas Children's Hospital & Baylor College of Medicine

Houston, Texas, 77030

United States

Vermont Children's Hospital & University of Vermont Medical Center

Burlington, Vermont, 05401

United States

Virginia Commonwealth University

Richmond, Virginia, 23298

United States

Seattle Children's Hospital

Seattle, Washington, 98105

United States

University of Wisconsin

Madison, Wisconsin, 53792

United States

Children's Wisconsin & Medical College of Wisconsin

Milwaukee, Wisconsin, 53226

United States

Collaborators and Investigators

Sponsor: University of Washington, the Collaborative Health Studies Coordinating Center

D. B. Sanders, MD, MS, PRINCIPAL_INVESTIGATOR, Indiana University
Margaret Rosenfeld, MD, MPH, PRINCIPAL_INVESTIGATOR, Seattle Children's Research Institute

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-12-01

Study Completion Date2029-02-28

Study Record Updates

Study Start Date2024-12-01

Study Completion Date2029-02-28

Terms related to this study

Keywords Provided by Researchers

cystic fibrosis
oral antibiotics
pulmonary exacerbation
pediatric

Additional Relevant MeSH Terms

Cystic Fibrosis