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

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

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.

Study Overview

• Status: Recruiting
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Other: Immediate Oral Antibiotics; Other: Tailored Treatment: Oral Antibiotics only if Additional Treatment needed

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)

• Study Type: Interventional
• Enrollment (Estimated): 430
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Erika Enright; Phone Number: 206-897-1922; Email: eenright@uw.edu

Study Locations

• Canada
  • British Columbia
    • Vancouver, British Columbia, Canada, V6H 3V4
      • Recruiting
      • British Columbia Children's Hospital
      • Principal Investigator: Jonathan Rayment, MD
      • Contact: Madison Weir; Email: Madison.Weir@bcchr.ca
  • Ontario
    • Toronto, Ontario, Canada, M5G 0A4
      • Recruiting
      • The Hospital for Sick Children & Toronto Canada CF Centre Pediatrics
      • Principal Investigator: Felix Ratjen, MD
      • Contact: Claire Crompton; Email: claire.crompton@sickkids.ca
• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Recruiting
      • The Children's Hospital Alabama & University of Alabama at Birmingham
      • Principal Investigator: Christopher Fowler, MD
      • Contact: Ashlyn Hastings; Email: aehastings@uabmc.edu
  • Arizona
    • Tucson, Arizona, United States, 85713
      • Recruiting
      • Tucson Cystic Fibrosis Center
      • Principal Investigator: Cori Daines, MD
      • Contact: Elizabeth (Lisa) Ryan; Email: elizabethryan@arizona.edu
  • California
    • Los Angeles, California, United States, 90027
      • Recruiting
      • Children's Hospital of Los Angeles & Anton Yelchin Cystic Fibrosis Clinic
      • Principal Investigator: Martha McKinney, MD
      • Contact: Jared Lopez, BA; Email: jalopez@chla.usc.edu
    • Palo Alto, California, United States, 94304
      • Recruiting
      • Stanford University
      • Contact: Tina Conti, BSRC, RRT, RRT-NPS, C-NPT; Email: tconti@stanford.edu
      • Principal Investigator: Lori WaiHang Lee, MD
    • San Diego, California, United States, 92123
      • Not yet recruiting
      • Rady Children's Hospital at University of California San Diego
      • Contact: Lisa Ramos, CRC; Email: lramosvallejo@health.ucsd.edu
      • Principal Investigator: Kathryn Akong, MD
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Recruiting
      • Children's Hospital of Colorado
      • Principal Investigator: Jordana Hoppe, MD
      • Contact: Mary Cross; Email: Mary.Cross@childrenscolorado.org
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Recruiting
      • Children's Healthcare of Atlanta & Emory University
      • Contact: Joy Dangerfield; Email: jdanger@emory.edu
      • Principal Investigator: Ajay Kasi, MD
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Not yet recruiting
      • Ann & Robert H. Lurie Children's Hospital of Chicago & Northwestern University
      • Principal Investigator: Maria Dowell, MD
      • Contact: Mariah Flowers; Email: maflowers@luriechildrens.org
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Recruiting
      • Riley Hospital for Children & Indiana University
      • Contact: Lisa Bendy; Email: lbendy@iu.edu
      • Principal Investigator: D. B. Sanders, MD, MS
      • Contact: Misty Thompson; Email: misthomp@iu.edu
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • Recruiting
      • University of Iowa
      • Contact: Mary Teresi; Email: mary-teresi@uiowa.edu
      • Principal Investigator: Anthony Fischer, MD
  • Maryland
    • Baltimore, Maryland, United States, 21287
      • Recruiting
      • Johns Hopkins Hospital, Johns Hopkins University
      • Contact: Jamelia Maynard; Email: jmaynar8@jhmi.edu
      • Principal Investigator: Peter Mogayzel, MD, PhD, MBA
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Recruiting
      • Boston Children's Hospital & Harvard University
      • Contact: Monica Ulles; Email: monica.ulles@childrens.harvard.edu
      • Principal Investigator: Gregory Sawicki, MD
  • Michigan
    • Ann Arbor, Michigan, United States, 48109-5212
      • Recruiting
      • University of Michigan Health System
      • Contact: Dawn Kruse; Email: dmkruse@med.umich.edu
      • Principal Investigator: Samya Nasr, MD
  • Minnesota
    • Minneapolis, Minnesota, United States, 55404
      • Recruiting
      • Children's Hospitals and Clinics of Minnesota
      • Principal Investigator: Brooke Moore, MD
      • Contact: Danniella Balangoy; Email: Danniella.Balangoy@childrensmn.org
    • Minneapolis, Minnesota, United States, 55455
      • Recruiting
      • The Minnesota Cystic Fibrosis Center & University of Minnesota
      • Principal Investigator: Samuel Goldfarb, MD
      • Contact: Alyssa Perry; Email: ahperry@umn.edu
  • Missouri
    • Kansas City, Missouri, United States, 64108
      • Recruiting
      • Children's Mercy Hospital
      • Contact: Jana Lomonte; Email: jblomonte@cmh.edu
      • Principal Investigator: Hugo Escobar, MD
    • St Louis, Missouri, United States, 63110
      • Recruiting
      • St. Louis Children's Hospital & Washington University School of Medicine
      • Principal Investigator: Jessica Pittman, MD
      • Contact: Emily Schulte, RN, BSN; Email: e.burns@wustl.edu
  • New York
    • Rochester, New York, United States, 14642
      • Recruiting
      • University of Rochester Medical Center Strong Memorial
      • Contact: Barbara Johnson, RN; Email: Barbara_johnson@urmc.rochester.edu
      • Principal Investigator: Matthew McGraw, MD
      • Contact: Karen McCarthy; Email: karen_mccarthy@urmc.rochester.edu
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • Recruiting
      • University of North Carolina at Chapel Hill
      • Principal Investigator: Jennifer Goralski, MD
      • Contact: Caroline Flowers, CRC; Email: caroline_flowers@med.unc.edu
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Recruiting
      • Cincinnati Children's Hospital Medical Center
      • Contact: Sharon Kadon, RN, BSN, CPN; Email: sharon.kadon@cchmc.org
      • Principal Investigator: Gary McPhail, MD
  • Oregon
    • Portland, Oregon, United States, 97239-3098
      • Recruiting
      • Oregon Health & Science University
      • Contact: Pierce Nusbaum; Email: nusbaum@ohsu.edu
      • Principal Investigator: Kelvin MacDonald, MD
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • Children's Hospital of Philadelphia & University of Pennsylvania
      • Principal Investigator: Clement Ren, MD, MBA
      • Contact: Matthew Gari; Email: garim@chop.edu
      • Contact: Erin Donnelly; Email: donnellye4@chop.edu
    • Pittsburgh, Pennsylvania, United States, 15224
      • Recruiting
      • Children's Hospital of Pittsburgh of UPMC & University of Pittsburgh Medical Center
      • Principal Investigator: Daniel Weiner, MD
      • Contact: Adrienne DeRicco, RN; Email: adrienne.dericco2@upmc.edu
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Recruiting
      • Medical University of South Carolina
      • Contact: Audra Wiser; Email: wisera@musc.edu
      • Principal Investigator: Sylvia E Szentpeter, MD
  • Texas
    • Dallas, Texas, United States, 75235
      • Recruiting
      • University of Texas Southwestern & Children's Health
      • Principal Investigator: Preeti Sharma, MD
      • Contact: Lindsay Allen, CRC; Email: Lindsay.Allen@utsouthwestern.edu
    • Houston, Texas, United States, 77030
      • Recruiting
      • Texas Children's Hospital & Baylor College of Medicine
      • Principal Investigator: Fadel Ruiz, MD
      • Contact: Pavel Tuekam; Email: OrnellaPavel.TuekamMeko@bcm.edu
  • Vermont
    • Burlington, Vermont, United States, 05401
      • Recruiting
      • Vermont Children's Hospital & University of Vermont Medical Center
      • Contact: Vanessa Marascio; Email: Vanessa.Marascio@uvmhealth.org
      • Principal Investigator: Thomas Lahiri, MD
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Recruiting
      • Virginia Commonwealth University
      • Contact: Akilah Pierre-Louis, CRC; Email: Akilah.PierreLouis1@vcuhealth.org
      • Principal Investigator: Andrew T. Barber, MD
  • Washington
    • Seattle, Washington, United States, 98105
      • Recruiting
      • Seattle Children's Hospital
      • Principal Investigator: Margaret Rosenfeld, MD
      • Contact: Mey Lee; Email: mey.lee@seattlechildrens.org
  • Wisconsin
    • Madison, Wisconsin, United States, 53792
      • Recruiting
      • University of Wisconsin
      • Principal Investigator: Hara Levy, MD
      • Contact: Nikki Cole, BS, RRT, CCRC; Email: ndondlinger@wisc.edu
    • Milwaukee, Wisconsin, United States, 53226
      • Recruiting
      • Children's Wisconsin & Medical College of Wisconsin
      • Contact: Laura Roth, CCRC; Email: lroth@mcw.edu
      • Principal Investigator: Nicholas Antos, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion 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 HEMT 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

Exclusion Criteria:

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

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 Oral Antibiotics; Increase airway clearance and start 14 days of preselected oral antibiotics right away
Experimental: Tailored Therapy; Increased airway clearance alone, with addition of oral antibiotics for worsening symptoms or failure to improve	Other: Tailored Treatment: Oral Antibiotics only if Additional Treatment needed; Increase airway clearance and start preselected oral antibiotics later if symptoms get worse or do not get better according to prespecified criteria

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
One-year change in pulmonary function by spirometry-measured ppFEV1	Compare the difference in pulmonary function between arms by evaluating change in spirometry-measured percent predicted forced expiratory volume (ppFEV1). A spirometry test measures the amount of air a person can forcibly exhale after a deep breath (forced vital capacity, or FVC) and the amount of air they can exhale in one second (forced expiratory volume in one second, or FEV1). A lower measured value compared to the reference value indicates lung disease.	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
One-Year change in pulmonary function by LCI	Compare the difference in pulmonary function between arms by evaluating change in Lung Clearance Index (LCI). LCI increases when there is lung disease, which causes ventilation inhomogeneity. LCI is calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity [FRC]) required to reduce end-tidal nitrogen concentration to 1/40th of the original level.	1 year

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Recovery to baseline by ppFEV1 and CRISS following a PEx	Compare recovery to baseline between arms over the 28-day period after the first pulmonary exacerbation (PEx), by spirometry-measured percent predicted forced expiratory volume (ppFEV1), and after all pulmonary exacerbations, measured by CRISS symptom scores. The Chronic Respiratory Infection Symptom Score (CRISS) ranges from 0 to 100, with higher scores indicating more symptoms.	28 days
Recovery to baseline by Lung Clearance Index (LCI)	Compare recovery to baseline between arms over the 28-day period after the first pulmonary exacerbation (PEx) in LCI, and after all exacerbations for symptom scores. The Chronic Respiratory Infection Symptom Score (CRISS) ranges from 0 to 100, with higher scores indicating more symptoms.	28 days
Safety Endpoint 1: Cumulative oral antibiotic exposure	Compare cumulative oral antibiotic exposure in each treatment arm.	1 year
Safety Endpoint 2: Number of respiratory illnesses	Compare the number of respiratory illnesses reported over the study period in each treatment arm.	1 year
Safety Endpoint 3: Proportion of respiratory illnesses treated with antibiotics during PEx	Compare the proportion of respiratory illnesses treated with antibiotics during the 28-day pulmonary exacerbation (PEx) treatment period in each treatment arm.	28 days
Safety Endpoint 4: Number of PEx treated with IV antibiotics	Compare the number of pulmonary exacerbations (PEx) treated with IV antibiotics over the study period in each treatment arm.	1 year
Safety Endpoint 5: Proportion of PEx failing to recover to baseline symptoms at Day 28	Compare the proportion of pulmonary exacerbations (PEx) failing to recover to baseline symptoms at Day 28 in each treatment arm.	28 days
Safety Endpoint 6: Proportion of PEx with ppFEV1 below baseline at Day 28	Compare the proportion of pulmonary exacerbations (PEx) with spirometry-measured percent predicted forced expiratory volume (ppFEV1) below baseline at Day 28 in each treatment arm.	1 year
Safety Endpoint 7: Time from initial randomized PEx to next respiratory illness	Compare the time from initial randomized pulmonary exacerbation (PEx) to next respiratory illness in each treatment arm.	1 year
Safety Endpoint 8: Targeted adverse events (patient report of antibiotic side effects during PEx)	Compare the targeted adverse events (patient report of antibiotic side effects during pulmonary exacerbation period) in each treatment arm.	1 year
Safety Endpoint 9: Treatment-emergent CF microorganisms on clinical respiratory cultures	Compare treatment-emergent CF microorganisms on clinical respiratory cultures in each treatment arm.	1 year
Qualitative Assessment 1: Caregivers' experience managing study treatment	To understand benefits and challenges associated with assigned treatment through targeted qualitative interviews of caregivers we will describe caregivers' experience managing study treatment and report themes that emerge regarding caregivers' experiences.	1 year
Qualitative Assessment 2: Study treatment impact on child and family daily life	To understand benefits and challenges associated with assigned treatment through targeted qualitative interviews of caregivers we will evaluate study treatment impact on child and family daily life and report themes that emerge regarding caregivers' experiences.	1 year
Health care costs 1: Total estimated cost of antibiotics	Compare total estimated cost of antibiotics used over the course of the study between treatment arms.	1 year
Health care costs 2: Health care utilization	Compare health care utilization over the course of the study between treatment arms, including number of primary care visits and number of emergency room visits.	1 year
Health care costs 3: Parent work productivity measures (WPAI)	To compare parent Work Productivity and Activity Impairment (WPAI) measures between study arms. The WPAI includes four scores assessing absenteeism, presenteeism, work productivity loss (overall work impairment/absenteeism plus presenteeism), and activity impairment in the last seven days. Scores range from 0 to 100, with higher scores indicating greater impairment.	1 year
Preschool and non-HEMT Pilot Cohort Primary Feasibility	Estimate the proportion of preschool and non-HEMT (Highly Effective Modulator Therapy) participants randomized to the tailored therapy arm that has no antibiotic exposure in the 28 days following a pulmonary exacerbation (PEx).	28 days
Preschool and non-HEMT Pilot Cohort Safety Endpoint 1: ppFEV1 and LCI recovery	Compare percent predicted forced expiratory volume (ppFEV1) and Lung Clearance Index (LCI) recovery using Day 28 values compared to nearest pre-PEx baseline measure in both treatment arms.	28 days
Preschool and non-HEMT Pilot Cohort Safety Endpoint 2: Time to symptom recovery	Compare time to symptom recovery (number of days to recover to baseline CRISS score) between treatment arms. The Chronic Respiratory Infection Symptom Score (CRISS) ranges from 0 to 100, with higher scores indicating more symptoms.	28 days
Preschool and non-HEMT Pilot Cohort Safety Endpoint 3: Targeted adverse events	Compare targeted adverse events (participant report of antibiotic side effects during PEx period) between treatment arms.	28 days
Preschool and non-HEMT Pilot Cohort Safety Endpoint 4: Treatment-emergent CF microorganisms	Compare treatment-emergent CF microorganisms on clinical respiratory cultures between treatment arms.	28 days
Preschool and non-HEMT Pilot Cohort Safety Endpoint 5: Treatment with additional non-protocol assigned antibiotics	Compare treatment with additional non-protocol assigned antibiotics within the 28 days post-randomization between treatment arms.	28 days
Preschool and non-HEMT Pilot Cohort Safety Endpoint 6: Time to IV antibiotics following a randomized PEx	Compare time to IV antibiotics following a randomized PEx (using CF Foundation Patient Registry data, if available) between treatment arms.	28 days
Preschool and non-HEMT Pilot Cohort: Future Larger Clinical Trial Feasibility 1	Measure the proportion of approached patients that enrolls.	28 days
Preschool and non-HEMT Pilot Cohort: Future Larger Clinical Trial Feasibility 2	Measure the proportion of respiratory symptoms meeting randomization criteria.	28 days
Preschool and non-HEMT Pilot Cohort: Future Larger Clinical Trial Feasibility 3	Measure the proportion of respiratory symptoms meeting randomization criteria resulting in treatment initiation within assigned arm.	28 days
Preschool and non-HEMT Pilot Cohort: Future Larger Clinical Trial Feasibility 4	Through targeted qualitative interviews of caregivers we will describe caregivers' experience managing study treatment and report themes that emerge regarding caregivers' experiences in order to understand motivations, benefits and challenges associated with assigned treatment arm.	28 days
Clinic Throat Swab Substudy 1: Metagenomic Sequencing	Use quantitative polymerase chain reaction (qPCR) and metagenomic sequencing to quantitatively define throat swab microbiology at baseline, at exacerbation diagnosis, and after treatment. Metagenomic sequencing analyzes the genomes of all microorganisms in a sample, providing a broad overview of the ecosystem's composition. It can be used to study the diversity of bacteria, detect the abundance of microbes, and study unculturable microorganisms.	1 year
Clinic Throat Swab Substudy 2: Microbiota Measures	Correlate throat microbiota measures (both pre-treatment microbial taxonomic or functional gene abundances, and changes in those abundances) with clinical outcome measures within antibiotic treatments.	1 year
Home Throat and Nasal Swab Substudy 1: Feasibility of Home Collection of Nasal Swabs for Viral Testing and Illness Frequency	Assess the feasibility of home collection of nasal swabs for viral testing at the time of pulmonary exacerbation (PEx) and determine the frequency of viral illness at the time of PEx diagnosis.	28 days
Home Throat and Nasal Swab Substudy 2: Feasibility of Home Collection of Throat Swabs for Bacterial testing	Assess the feasibility of home collection of throat swabs for bacterial testing at the time of pulmonary exacerbation (PEx).	28 days
Home Throat and Nasal Swab Substudy 3: Association Between Viral Illness and Symptom recovery	Evaluate the association between the presence of viral illness and symptom recovery (number of days to recover to baseline CRISS score) between treatment arms. The Chronic Respiratory Infection Symptom Score (CRISS) ranges from 0 to 100, with higher scores indicating more symptoms.	28 days
Home Throat and Nasal Swab Substudy 3: Association Between Viral Illness and FEV1	Evaluate the association between the presence of viral illness and spirometry-measured forced expiratory volume (FEV1) at Day 28.	28 days
Home Throat and Nasal Swab Substudy 3: Association Between Viral Illness and antibiotic treatment.	Evaluate the association between the presence of viral illness and the percent of subjects in the tailored therapy arm requiring antibiotic treatment.	28 days
Home Throat and Nasal Swab Substudy 5: Bacterial Pathogen Identification, Frequency and Impact on Treatment Changes	Determine the concordance of bacteria identified on CF pathogen testing at the time of pulmonary exacerbation (PEx) compared to the most recent routine clinic visit. In addition, assess how frequently new bacterial pathogens are identified at the time of PEx and how this impacts treatment changes, including the need to change or add antibiotic therapy.	28 days
Remote Monitoring Substudy 1: Feasibility of Home Spirometry Use During PEx	Describe the feasibility and user acceptability of home spirometry during pulmonary exacerbations (PEx) in children with CF via survey of participants at the end of the study.	28 days
Remote Monitoring Substudy 2: Feasibility of BioButton Use During PEx	Describe the feasibility and user acceptability of BioButton use during pulmonary exacerbations in children with CF via survey of participants at the end of the study.	14 days
Remote Monitoring Substudy 3: Change in FEV1 by Home Spirometry	Describe the change from nearest baseline-state FEV1 to Day 0 exacerbation state measurement and the trajectory of change during the exacerbation state (through Day 28).	28 days
Remote Monitoring Substudy 4: Compare Home Spirometry vs. Clinic Spirometry Measures	Compare measurements and their change measured by home spirometry vs. clinic spirometry during the exacerbation state.	28 days
Remote Monitoring Substudy 5: BioButton-measured Change in Resting Heart Rate During PEx	Describe changes in resting heart rate from Day 0 to 14 of the exacerbation state.	14 days
Remote Monitoring Substudy 5: BioButton-measured Change in Resting respiratory rate During PEx	Describe changes in Resting respiratory rate from Day 0 to 14 of the exacerbation state.	14 days
Remote Monitoring Substudy 5: BioButton-measured Change in Activity Level During PEx	Describe changes activity level, measured using actigraphy, and summarized as the mean (SD) number of active minutes in a day, from Day 0 to 14 of the exacerbation state.	14 days
Remote Monitoring Substudy 5: BioButton-measured Change in Sleep During PEx	Describe changes in sleep time from Day 0 to 14 of the exacerbation state.	14 days
Remote Monitoring Substudy 6: Compare BioButton-measured Change in resting heart rate During PEx with Baseline Averages	Compare changes in resting HR to averages from a 7-day period during baseline state.	14 days
Remote Monitoring Substudy 6: Compare BioButton-measured Change in Resting respiratory rate During PEx with Baseline Averages	Compare changes in BioButton-measured Resting respiratory rate to averages from a 7-day period during baseline state.	14 days
Remote Monitoring Substudy 6: Compare BioButton-measured Change in activity level During PEx with Baseline Averages	Compare BioButton-measured change in activity level during pulmonary exacerbation (PEx) to averages from a 7-day period during baseline state.	14 days
Remote Monitoring Substudy 6: Compare BioButton-measured Change in Sleep During PEx with Baseline Averages	Compare BioButton change in sleep during PEx to averages from a 7-day period during baseline state.	14 days
Remote Monitoring Substudy 7: Determine correlations between changes in FEV1 and CRISS to Resting Heart Rate (HR)	Determine correlations between changes from Day 0 to 14 in forced expiratory volume (FEV1) and CRISS score to Resting Heart Rate (HR). The Chronic Respiratory Infection Symptom Score (CRISS) ranges from 0 to 100, with higher scores indicating more symptoms.	14 days
Remote Monitoring Substudy 7: Determine correlations between changes in FEV1 and CRISS to Resting Respiratory rate	Determine correlations between changes from Day 0 to 14 in forced expiratory volume (FEV1) and CRISS score to Resting Respiratory rate. The Chronic Respiratory Infection Symptom Score (CRISS) ranges from 0 to 100, with higher scores indicating more symptoms.	14 days
Remote Monitoring Substudy 7: Determine correlations between changes in FEV1 and CRISS to Activity level	Determine correlations between changes from Day 0 to 14 in forced expiratory volume (FEV1) and CRISS score to Activity level, measured with actigraphy. The Chronic Respiratory Infection Symptom Score (CRISS) ranges from 0 to 100, with higher scores indicating more symptoms.	14 days
Remote Monitoring Substudy 7: Determine correlations between changes in FEV1 and CRISS to Sleep duration	Determine correlations between changes from Day 0 to 14 in forced expiratory volume (FEV1) and CRISS score to sleep duration. The Chronic Respiratory Infection Symptom Score (CRISS) ranges from 0 to 100, with higher scores indicating more symptoms.	14 days

Collaborators and Investigators

• Sponsor: University of Washington, the Collaborative Health Studies Coordinating Center
• Collaborators: Cystic Fibrosis Foundation
• Investigators: Principal Investigator: Margaret Rosenfeld, MD, MPH, Seattle Children's Hospital; Principal Investigator: D. B. Sanders, MD, MS, Indiana University

Publications and helpful links

General Publications

• 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.

Helpful Links

• STOP PEDS Pilot Study manuscript

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2024
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): February 28, 2029

Study Registration Dates

• First Submitted: October 14, 2024
• First Submitted That Met QC Criteria: October 21, 2024
• First Posted (Actual): October 23, 2024

Study Record Updates

• Last Update Posted (Actual): May 14, 2026
• Last Update Submitted That Met QC Criteria: May 12, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

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

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