Symptom driven for Cystic Fibrosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
University of Missouri Hospital and Clinics, Columbia, MO
Cystic Fibrosis+2 More
Symptom driven performance of airway clearance - Other
Eligibility
18+
All Sexes
What conditions do you have?
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Study Summary

Cystic Fibrosis (CF) is an autosomal recessive disease cause by a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) manifesting in multiple organs, the most common cause of morbidity and mortality continues to be the pulmonary manifestation. CFTR dysfunction leads to reduced mucociliary clearance, impaired innate immune system function in the lungs (within the airway surface liquid [ASL] lining the epithelial barrier of the lungs) and reduced ASL hydration (stickier mucus). To try and help correct this underlying defect patients have been performing airway clearance for decades using different techniques (Percussion and postural drainage [P&PD], Positive expiratory pressure [PEP], Oscillatory positive expiratory pressure [OPEP], High-frequency chest compression [HFCC], exercise), inhaled mucolytics (Hypertonic Saline, Pulmozyme) and inhaled antibiotics. However, performing daily airway clearance can be a large burden on patients and their families with a median number of daily therapies around 7 and average time spent on therapies at almost 2 hours daily. This high treatment burden leads many patients to have reduced adherence to their regimens and multiple studies have shown around 20% of patients performing no daily airway clearance. Since the release of highly effective CFTR modulator therapy patients have experienced improvements in lung function measurements and imaging-based ventilation measurements, reduction in pulmonary exacerbations, and improvement in daily symptom scores. Over 80% of patients and their families and over 95% of clinicians in the United States support the idea of trials looking into the simplification of airway clearance regimens. Combining the inability of most patients to complete their daily regimens, patient and clinician interest in treatment simplification research, and the overwhelming cost of most inhaled medications in cystic fibrosis with the improvement in mucociliary transport and symptoms with highly effective modulator therapy suggests a research program aimed at reducing the treatment burden of daily airway clearance should be considered. The investigators propose the following: determine if there is additional benefit in continuous airway clearance regimens after starting Elexacaftor-Tezacaftor-Ivacaftor (ETI) and if so, is this benefit noticeable on pulmonary function testing and imaging.

Eligible Conditions

  • Cystic Fibrosis
  • Mucociliary Clearance Defect

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Cystic Fibrosis

Study Objectives

3 Primary · 4 Secondary · Reporting Duration: 12 weeks

12 weeks
Absolute change in MRI xenon gas ventilation defect scoring from week 0 to week 12 in symptom driven airway clearance arm
Absolute change in percent predicted FEV1 (ppFEV1) from week 0 to week 12 in symptom driven airway clearance arm
Absolute change in percent predicted FVC (ppFVC) from week 0 to week 12 in symptom driven airway clearance arm
Absolute change in respiratory symptoms based upon Cystic Fibrosis Questionnaire Revise score
Change in airway colonization with S. aureus or P. aeruginosa
Frequency of performance of airway clearance in symptom driven airway clearance arm
Incidence of adverse events (AE) in the symptom driven airway clearance arm

Trial Safety

Safety Progress

1 of 3

Other trials for Cystic Fibrosis

Trial Design

2 Treatment Groups

Continuous
1 of 2
Symptom driven
1 of 2
Active Control
Experimental Treatment

40 Total Participants · 2 Treatment Groups

Primary Treatment: Symptom driven · No Placebo Group · N/A

Symptom driven
Other
Experimental Group · 1 Intervention: Symptom driven performance of airway clearance · Intervention Types: Other
Continuous
Other
ActiveComparator Group · 1 Intervention: Continuous daily performance of airway clearance · Intervention Types: Other

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 12 weeks

Trial Background

Prof. Zach Holliday, Associate Professor
Principal Investigator
University of Missouri-Columbia
Closest Location: University of Missouri Hospital and Clinics · Columbia, MO
Photo of columbia  1Photo of columbia  2Photo of columbia  3
2012First Recorded Clinical Trial
1 TrialsResearching Cystic Fibrosis
10 CompletedClinical Trials

Eligibility Criteria

Age 18+ · All Participants · 4 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You must be at least 18 years old at the time of recruitment.
You have no exacerbations in the last 28 days.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.

References