Cystic FibrosisSTOP-PEDSNCT04608019
Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Pilot Study
A study testing Immediate Antibiotics and Tailored Treatment in people with Cystic Fibrosis, run by University of Washington, the Collaborative Health Studies Coordinating Center at 10 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Immediate Antibiotics and Tailored Treatment
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 121 participants
- Where
- 10 sitesUnited States
- Study dates
- 10 Nov 2020to 18 Aug 2022
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Enrollment Inclusion Criteria:
- 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
Enrollment Exclusion Criteria:
- 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
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Children's Hospital of Colorado
Aurora, Colorado, 80045
Lurie Children's Hospital of Chicago & Northwestern University
Chicago, Illinois, 60611-2605
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Texas Children's Hospital and Baylor College of Medicine
Houston, Texas, 77030
Riley Hospital for Children
Indianapolis, Indiana, 46202
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Oregon Health Sciences University
Portland, Oregon, 97239
Seattle Children's Hospital
Seattle, Washington, 98105
Tucson Cystic Fibrosis Center
Tucson, Arizona, 85713
About this study
Official title: Streamlined Treatment of Pulmonary Exacerbations in Pediatrics (Pilot)
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.
Read the 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.
- Conditions
- Cystic Fibrosis
- Being tested
- Immediate Antibiotics, Tailored Treatment
- Keywords
- cystic fibrosis, oral antibiotics, pulmonary exacerbation, pediatric
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Immediate AntibioticsOther
increase airway clearance/start oral antibiotics right away
Tailored TreatmentOther
increase airway clearance and start oral antibiotics later if symptoms get worse or do not get better
| Study group | Role | What they receive |
|---|---|---|
| Immediate Antibiotics | Experimental | increased airway clearance plus early initiation of oral antibiotics Receives: Other: Immediate Antibiotics |
| Tailored Therapy | Experimental | increased airway clearance alone, with addition of antibiotics for worsening symptoms or failure to improve Receives: Other: Tailored Treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
Delayed antibiotics
The proportion of participants in the tailored arm who did not take any oral antibiotics in the 28 days following randomization
Measured: 28 days
Secondary outcomes (6)
Consent
Proportion of approached patients consenting to enroll
Measured: 6 months
Pulmonary Exacerbations Reported
Proportion of Pulmonary Exacerbations in which symptoms are reported within 7 days of onset
Measured: 18 months
Randomization Criteria
Proportion of Pulmonary Exacerbation events meeting randomization criteria
Measured: 18 months
Participant Exacerbations
Proportion of enrolled participants experiencing a randomizable Pulmonary Exacerbation
Measured: 18 months
Randomized Exacerbations
Proportion of randomizable Pulmonary Exacerbations that undergo randomization
Measured: 18 months
Day 28 Follow-up
Proportion of participants with a randomized Pulmonary Exacerbation that attends an in-person Day 28 follow up visit
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2020
- Study start10 Nov 2020
- Primary completion18 Aug 2022
- Study completion18 Aug 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 121 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Washington, the Collaborative Health Studies Coordinating Center
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Sponsor
- Investigators
- Donald B. Sanders, MD, Principal investigator, Riley Children's Hospital, Indianapolis, INMargaret Rosenfeld, MD, Principal investigator, Seattle Children's Hospital, Seattle, WA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04608019
- Sponsor's protocol ID
- STOP-PEDS
- Acronym
- STOP-PEDS
- Status last verified
- Oct 2024
- First submitted
- 23 Oct 2020
- Last updated
- 18 Oct 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 11401068Sanders 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.
PubMed 37683122
Study at a glance
From the registry record, last updated by the sponsor on 18 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Streamlined Treatment of Pulmonary Exacerbations in Pediatrics (Pilot)
- ClinicalTrials.gov ID
- NCT04608019
- Acronym / sponsor ID
- STOP-PEDS / STOP-PEDS
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, oral antibiotics, pulmonary exacerbation, pediatric
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 121 participants (actual)
- Lead sponsor
- University of Washington, the Collaborative Health Studies Coordinating CenterUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- 10 Nov 2020 (actual)
- Primary completion
- 18 Aug 2022 (actual)
- Study completion
- 18 Aug 2022 (actual)
- First posted
- 29 Oct 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Immediate Antibiotics | Experimental | Other: Immediate Antibioticsincreased airway clearance plus early initiation of oral antibiotics |
| Tailored Therapy | Experimental | Other: Tailored Treatmentincreased airway clearance alone, with addition of antibiotics for worsening symptoms or failure to improve |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Immediate Antibioticsincrease airway clearance/start oral antibiotics right away | Other | Immediate Antibiotics |
| Tailored Treatmentincrease airway clearance and start oral antibiotics later if symptoms get worse or do not get better | Other | Tailored Therapy |
Primary outcome
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
6 secondary outcomes
Consent
Time frame: 6 months
Pulmonary Exacerbations Reported
Time frame: 18 months
Randomization Criteria
Time frame: 18 months
Participant Exacerbations
Time frame: 18 months
Randomized Exacerbations
Time frame: 18 months
Day 28 Follow-up
Time frame: 18 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Oct 2024.
- Background: PubMed 11401068Bradley 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.
- Results publication: PubMed 37683122Sanders 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.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Donald B. Sanders, MD principal investigator
Riley Children's Hospital, Indianapolis, IN
Margaret Rosenfeld, MD principal investigator
Seattle Children's Hospital, Seattle, WA
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Immediate Antibiotics and Tailored Treatment in people with Cystic Fibrosis, run by University of Washington, the Collaborative Health Studies Coordinating Center at 10 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Delayed antibiotics (time frame: 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04608019, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Oct 2024.