Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Pilot Study

NCT04608019 | Completed DMC

• 1 other identifier: STOP-PEDS
• Study Type: interventional
• Target: 121
• Locations: 1 country
• Sites: 10

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.

Conditions

Cystic Fibrosis

Keywords

cystic fibrosis; oral antibiotics; pulmonary exacerbation; pediatric

Outcome Measures

Primary Outcomes (1)

• Delayed antibiotics

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

  28 days

Secondary Outcomes (6)

• Consent

  6 months

• Pulmonary Exacerbations Reported

  18 months

• Randomization Criteria

  18 months

• Participant Exacerbations

  18 months

• Randomized Exacerbations

  18 months

Study Arms (2)

Immediate Antibiotics

EXPERIMENTAL

increased airway clearance plus early initiation of oral antibiotics

Other: Immediate Antibiotics

Tailored Therapy

EXPERIMENTAL

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

Other: Tailored Treatment

Interventions

Immediate Antibiotics OTHER

increase airway clearance/start oral antibiotics right away

Immediate Antibiotics

Tailored Treatment OTHER

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

Tailored Therapy

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Washington, the Collaborative Health Studies Coordinating Center: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (10)

Tucson Cystic Fibrosis Center

Tucson, Arizona, 85713, United States

Location

Children's Hospital of Colorado

Aurora, Colorado, 80045, United States

Location

Children's Healthcare of Atlanta

Atlanta, Georgia, 30322, United States

Location

Lurie Children's Hospital of Chicago & Northwestern University

Chicago, Illinois, 60611-2605, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States

Location

Oregon Health Sciences University

Portland, Oregon, 97239, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

Texas Children's Hospital and Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Related Publications (2)

• 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.

  PMID: 11401068 BACKGROUND

• 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.

  PMID: 37683122 RESULT

Related Links

• Related Info

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Donald B. Sanders, MD

  Riley Children's Hospital, Indianapolis, IN

  PRINCIPAL INVESTIGATOR

• Margaret Rosenfeld, MD

  Seattle Children's Hospital, Seattle, WA

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 23, 2020
• First Posted: October 29, 2020
• Study Start: November 10, 2020
• Primary Completion: August 18, 2022
• Study Completion: August 18, 2022
• Last Updated: October 18, 2024

Record last verified: 2024-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)