Streamlined Treatment of Pulmonary Exacerbations in Pediatrics

Cystic Fibrosis Clinical Trial

— STOP-PEDS  

Official title:

Streamlined Treatment of Pulmonary Exacerbations in Pediatrics (Pilot)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04608019
• Other study ID #: STOP-PEDS
• Status: Completed
• Phase: N/A
• Start date: November 10, 2020
• Est. completion date: August 18, 2022

Study information

• Verified date: March 2024
• Source: University of Washington, the Collaborative Health Studies Coordinating Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 121
• Est. completion date: August 18, 2022
• Est. primary completion date: August 18, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 18 Years

Eligibility

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 or IV antibiotics for respiratory symptoms since January 1, 2019.

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Other:
• Immediate Antibiotics
increase airway clearance/start oral antibiotics right away
• Tailored Treatment
increase airway clearance and start oral antibiotics later if symptoms get worse or do not get better

Locations

Country	Name	City	State
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Children's Hospital of Colorado	Aurora	Colorado
United States	Lurie Children's Hospital of Chicago & Northwestern University	Chicago	Illinois
United States	Helen DeVos Children's Hospital	Grand Rapids	Michigan
United States	Texas Children's Hospital and Baylor College of Medicine	Houston	Texas
United States	Riley Hospital for Children	Indianapolis	Indiana
United States	Children's Hospital of Pittsburgh of UPMC	Pittsburgh	Pennsylvania
United States	Oregon Health Sciences University	Portland	Oregon
United States	Seattle Children's Hospital	Seattle	Washington
United States	Tucson Cystic Fibrosis Center	Tucson	Arizona

Sponsors (2)

Lead Sponsor	Collaborator
University of Washington, the Collaborative Health Studies Coordinating Center	Cystic Fibrosis Foundation

Country where clinical trial is conducted

United States, 

References & 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. — View Citation

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 T — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Consent	Proportion of approached patients consenting to enroll	6 months
Secondary	Pulmonary Exacerbations Reported	Proportion of Pulmonary Exacerbations in which symptoms are reported within 7 days of onset	18 months
Secondary	Randomization Criteria	Proportion of Pulmonary Exacerbation events meeting randomization criteria	18 months
Secondary	Participant Exacerbations	Proportion of enrolled participants experiencing a randomizable Pulmonary Exacerbation	18 months
Secondary	Randomized Exacerbations	Proportion of randomizable Pulmonary Exacerbations that undergo randomization	18 months
Secondary	Day 28 Follow-up	Proportion of participants with a randomized Pulmonary Exacerbation that attends an in-person Day 28 follow up visit	18 months

External Links

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