Streamlined Treatment of Pulmonary Exacerbations in Pediatrics
Start Date
12/1/2024
Completion Date
2/28/2029
Summary
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.
Detailed Description
The STOP PEDS pilot study demonstrated that a randomized trial of treatment strategies for mild pulmonary exacerbations (PEx) in children with CF was feasible and that assignment to a tailored therapy arm (defined below) may reduce antibiotic exposure. Based on the research priorities identified by CF families and clinicians and the results of the pilot study, 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 PEx in the pediatric CF population. The two treatment arms are immediate antibiotics and tailored therapy. In both arms, participants will be instructed to increase airway clearance at the onset of an eligible PEx. In the immediate antibiotics arm, they will also begin 14 days of oral antibiotics preselected by their primary CF providers, while in the tailored therapy they will only begin antibiotics if prespecified criteria for worsening symptoms or failure to improve are met. The STOP PEDS study will enroll three cohorts. In the main cohort, children ages 6-18 on highly effective modulator therapy (HEMT) will be enrolled when well and followed for 12 months. Participants will be randomly assigned to a treatment arm and maintain that treatment assignment for all subsequent eligible PEx during their 12-month enrollment period. Two additional pilot cohorts, the preschool cohort (children ages 3 to 5 on HEMT) and the non-HEMT cohort (children ages 6-18 not eligible for HEMT), will be enrolled in parallel pilot studies. Participants will enroll when well and be followed through one randomized PEx. Participants in the STOP PEDS RCT at selected sites will have the opportunity to enroll in optional substudies if eligible. These substudies include: * Clinic throat swab substudy * Home throat and nasal swab substudy * Remote monitoring substudy (Home monitoring of lung function, vital signs, activity and sleep)
Eligibility Criteria
Age Range: 3 years to 18 years
Interventions
Immediate Oral Antibiotics
Tailored Treatment: Oral Antibiotics only if Additional Treatment needed
Conditions
Locations
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
St 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
British Columbia Children's Hospital
Vancouver, British Columbia V6H 3V4
Canada
The Hospital for Sick Children & Toronto Canada CF Centre Pediatrics
Toronto, Ontario M5G 0A4
Canada