430 Participants Needed

Antibiotic Treatments for Cystic Fibrosis

(STOP PEDS RCT Trial)

Recruiting at 33 trial locations
EE
Overseen ByErika Enright
Age: < 65
Sex: Any
Trial Phase: Academic
Sponsor: University of Washington, the Collaborative Health Studies Coordinating Center
Must be taking: ETI, Ivacaftor
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests two antibiotic approaches for children with cystic fibrosis (CF) experiencing lung flare-ups. The goal is to determine which method—starting antibiotics immediately (Immediate Oral Antibiotics) or using them only if symptoms worsen (Tailored Treatment: Oral Antibiotics only if Additional Treatment needed)—proves more effective and safer over a year. Children diagnosed with CF who can perform a simple lung test may qualify. As an unphased trial, this study provides a unique opportunity for participants to contribute to research that could enhance treatment strategies for CF.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications before enrolling. You cannot be on an acute course of oral or IV antibiotics or systemic corticosteroids within 14 days prior to enrollment, and you cannot be on chronic oral antibiotics other than azithromycin at enrollment.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that taking oral antibiotics immediately for cystic fibrosis (CF) often works well and is easy for patients to manage. One study found that a single course of these antibiotics helps manage lung flare-ups in CF patients. Another study discovered that 70% of patients didn't need antibiotics, suggesting many people do well without them initially. This indicates that antibiotics are generally safe when used early, but not everyone needs them right away.

For a more personalized treatment approach, where antibiotics are given only if necessary, studies have also shown good results. Patients start with increased airway clearance and receive antibiotics only if their symptoms worsen or don't improve. This method has been tested for safety and is considered a reasonable option for managing CF. Overall, both strategies have proven safe for treating CF in children.12345

Why are researchers excited about this trial's treatments?

Researchers are excited about these treatments for cystic fibrosis because they offer a more personalized approach compared to traditional methods, which often rely on a fixed schedule of antibiotics. The Immediate Antibiotics treatment provides early intervention with oral antibiotics and increased airway clearance, potentially reducing the severity of infections right from the start. Meanwhile, the Tailored Therapy focuses on increased airway clearance alone, with antibiotics added only if symptoms worsen, allowing for a more adaptable and potentially less antibiotic-heavy regimen. This flexible approach could minimize antibiotic resistance and side effects, addressing a key concern with long-term antibiotic use.

What evidence suggests that this trial's treatments could be effective for cystic fibrosis?

This trial compares two approaches to antibiotic treatment for cystic fibrosis. Participants in one arm receive Immediate Oral Antibiotics, which involves increased airway clearance and early initiation of oral antibiotics. Studies have shown that oral antibiotics effectively treat worsening lung symptoms in people with cystic fibrosis, improving both symptom severity and quality of life.

In the other arm, participants follow a Tailored Therapy approach, which involves increased airway clearance alone, adding oral antibiotics only if symptoms worsen or fail to improve. Research suggests that this personalized treatment plan allows for a more tailored approach to managing symptoms. Overall, both immediate and tailored antibiotic treatments effectively manage cystic fibrosis symptoms.12367

Who Is on the Research Team?

DB

D. B. Sanders, MD, MS

Principal Investigator

Indiana University

MR

Margaret Rosenfeld, MD, MPH

Principal Investigator

Seattle Children's Hospital

Are You a Good Fit for This Trial?

This trial is for children and teens with Cystic Fibrosis who can give consent, use text messaging and the internet, perform spirometry tests, and meet specific health criteria. It's not for those recently on antibiotics or steroids, with certain infections or organ transplants, or on chronic oral antibiotics.

Inclusion Criteria

Written informed consent obtained from participant or participant's legal representative and ability to comply with study requirements
Ability to receive text messages and access the internet
I can successfully complete lung function tests.
See 3 more

Exclusion Criteria

Positive culture for Mycobacterium abscessus in the 12 months prior to enrollment
Presence of a condition compromising safety or data quality
I have recently taken antibiotics or steroids.
See 5 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive either immediate antibiotics or tailored therapy for pulmonary exacerbations, with increased airway clearance.

14 days per exacerbation
Regular monitoring visits as needed

Follow-up

Participants are monitored for safety and effectiveness after treatment, including assessment of pulmonary function and adverse events.

1 year
Periodic in-person and remote monitoring visits

Optional Substudies

Participants may enroll in optional substudies for additional monitoring and data collection, such as throat swabs and remote monitoring.

Varies by substudy

What Are the Treatments Tested in This Trial?

Interventions

  • Immediate Oral Antibiotics
  • Tailored Treatment: Oral Antibiotics only if Additional Treatment needed
Trial Overview The STOP PEDS RCT compares two antibiotic treatments for managing pulmonary exacerbations in kids with CF: one starts oral antibiotics only if needed later; the other begins immediate oral antibiotics. The study observes long-term (one year) and short-term safety and effectiveness.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Group I: Tailored TherapyExperimental Treatment1 Intervention
Group II: Immediate AntibioticsExperimental Treatment1 Intervention

Immediate Oral Antibiotics is already approved in European Union, United States, Canada for the following indications:

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Approved in European Union as Immediate Oral Antibiotics for:
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Approved in United States as Immediate Oral Antibiotics for:
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Approved in Canada as Immediate Oral Antibiotics for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Washington, the Collaborative Health Studies Coordinating Center

Lead Sponsor

Trials
7
Recruited
10,100+

Cystic Fibrosis Foundation

Collaborator

Trials
199
Recruited
37,800+

Published Research Related to This Trial

Pediatric care sites with better pulmonary function (highest quartile) were more likely to prescribe antibiotics for pulmonary exacerbations (PEx) in cystic fibrosis patients, with a significant increase in treatment odds compared to lower quartile sites.
The study found that the likelihood of receiving antibiotic treatment increased with the severity of PEx symptoms, suggesting that timely antibiotic intervention may contribute to better pulmonary function outcomes.
Antibiotic treatment of signs and symptoms of pulmonary exacerbations: a comparison by care site.Schechter, MS., Regelmann, WE., Sawicki, GS., et al.[2018]
A systematic review of four trials involving 167 participants found insufficient high-quality evidence to determine the effectiveness of inhaled antibiotics compared to intravenous antibiotics for treating pulmonary exacerbations in cystic fibrosis.
No significant differences were observed in quality of life, lung function, or the need for additional antibiotics between inhaled and intravenous treatments, indicating a need for further research to clarify the role of inhaled antibiotics in this context.
Inhaled antibiotics for pulmonary exacerbations in cystic fibrosis.Smith, S., Rowbotham, NJ., Charbek, E.[2022]
In a study of 312 children with cystic fibrosis, 763 oral antibiotic courses were prescribed, with 58% initiated over the phone, highlighting the importance of telemedicine in managing pulmonary exacerbations.
Nineteen percent of patients were hospitalized within three months after receiving oral antibiotics, but 87% showed recovery in lung function (FEV1) to near baseline levels within six months, indicating that while oral antibiotics are effective, there is still a significant risk of hospitalization.
Oral antibiotic prescribing patterns for treatment of pulmonary exacerbations in two large pediatric CF centers.Hoppe, JE., Hinds, DM., Colborg, A., et al.[2021]

Citations

Characteristics and outcomes of oral antibiotic treated ...Oral antibiotic treatment was associated with a significant improvement in exacerbation score and quality of life measured by the CF ...
Update on the diagnosis and management of cystic fibrosis ...In the end, results from this study indicate that extending IV antibiotics beyond 14 days does not improve lung function and that in some people, 10 days of IV ...
Oral Antimicrobial Use in Outpatient Cystic Fibrosis ...Our observations suggest that one course of oral antimicrobials is frequently effective in outpatient CF pulmonary exacerbations.
A Pilot Randomized Clinical Trial of Pediatric Cystic ...Effect of pulmonary exacerbations treated with oral antibiotics on clinical outcomes in cystic fibrosis. Thorax 2017;72:327–332. Crossref ...
Inhaled antibiotics for pulmonary exacerbations in cystic ...Inhaled antibiotics plus intravenous antibiotics may make little or no difference to quality of life compared to intravenous antibiotics alone.
Antibiotic Therapy for Pulmonary Exacerbations in Cystic ...This prospective single-center study (DRKS00012924) analyzes exacerbation treatment over 28 days in 96 pediatric and adult people with cystic fibrosis.
Streamlined Treatment of Pulmonary Exacerbations in ...In the immediate antibiotics arm, they will also begin 14 days of oral antibiotics ... Cystic Fibrosis Pulmonary Exacerbations Treatment Strategies. Ann Am ...
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