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

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ClinicalTrials.gov Identifier: NCT04608019
Recruitment Status : Recruiting
First Posted : October 29, 2020
Last Update Posted : January 15, 2021
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
University of Washington, the Collaborative Health Studies Coordinating Center

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 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


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




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



Information from the National Library of Medicine

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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 antibiotics for respiratory symptoms in the 12 months prior to enrollment
  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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04608019


Contacts
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Contact: Traci Bartz, MS (206) 897-1904 bartzt@uw.edu
Contact: Erika Enright (206) 897-1922 eenright@uw.edu

Locations
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United States, Arizona
Tucson Cystic Fibrosis Center Recruiting
Tucson, Arizona, United States, 85713
Contact: Elizabeth (Lisa) Ryan    520-626-3125    elizabethryan@arizona.edu   
Contact: Monica Teresa Varela    520-626-0462    monicavarela@arizona.edu   
Principal Investigator: Cori Daines, MD         
United States, Colorado
Children's Hospital of Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Mary Cross    720-777-4645    Mary.Cross@childrenscolorado.org   
Contact: Dana Coyle    720-777-6162    dana.coyle@childrenscolorado.org   
Principal Investigator: Jordana Hoppe, MD         
United States, Georgia
Children's Healthcare of Atlanta Recruiting
Atlanta, Georgia, United States, 30322
Contact: Akele Carter    404-785-9011    Akele.carter@choa.org   
Contact: Ashleigh Streby    404-727-0305    ashleigh.streby@emory.edu   
Principal Investigator: Kevin Kirchner, MD         
United States, Illinois
Lurie Children's Hospital of Chicago & Northwestern University Recruiting
Chicago, Illinois, United States, 60611-2605
Contact: Trecia Dawkins    312-227-1258    tdawkins@luriechildrens.org   
Contact: Larissa Rugg    312-227-6865    lrugg@luriechildrens.org   
Principal Investigator: Susanna McColley, MD         
United States, Indiana
Riley Hospital for Children Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Misty Thompson, B.S.    317-274-7768    misthomp@iupui.edu   
Contact: Lisa Bendy    317-948-7152    lbendy@iu.edu   
Principal Investigator: D. B. Sanders, MD         
United States, Michigan
Helen DeVos Children's Hospital Recruiting
Grand Rapids, Michigan, United States, 49503
Contact: Cindy Gile    616-391-6148    Cynthia.Gile@spectrumhealth.org   
Contact: Melisa Enos    616-486-6082    Melisa.Enos@spectrumhealth.org   
Principal Investigator: Susan Millard, MD         
United States, Oregon
Oregon Health Sciences University Recruiting
Portland, Oregon, United States, 97239
Contact: Annie Hanson    503-494-5487    hansoan@ohsu.edu   
Contact: Brendan Klein    503-418-8108    kleinb@ohsu.edu   
Principal Investigator: Kelvin MacDonald, MD         
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Adrienne DeRicco    412-692-8069    Adrienne.Dericco2@upmc.edu   
Contact: Elizabeth Hartigan    412-692-7060    elizabeth.hartigan@chp.edu   
Principal Investigator: Daniel Weiner, MD         
United States, Texas
Texas Children's Hospital and Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: Roger Hernandez    832-822-3389    rxherna2@texaschildrens.org   
Principal Investigator: Fadel Ruiz, MD         
United States, Washington
Seattle Children's Hospital Recruiting
Seattle, Washington, United States, 98105
Contact: Sharon McNamara    206-987-3921    sharon.mcnamara@seattlechildrens.org   
Contact: Sabrina Gorry    206-987-3921    sabrina.gorry@seattlechildrens.org   
Principal Investigator: Margaret Rosenfeld, MD         
Sponsors and Collaborators
University of Washington, the Collaborative Health Studies Coordinating Center
Cystic Fibrosis Foundation
Investigators
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Principal Investigator: Donald B. Sanders, MD Riley Children's Hospital, Indianapolis, IN
Principal Investigator: Margaret Rosenfeld, MD Seattle Children's Hospital, Seattle, WA
Publications:
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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    Key Record Dates
Last Update Posted: January 15, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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:
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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