A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis

Study Overview
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Brief Summary
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This phase IV, multicenter, randomized, open-label, two-period crossover study will evaluate the comparable efficacy and safety of Pulmozyme (dornase alfa) delivered by the eRapid nebulizer system in patients with cystic fibrosis. Patients who have been receiving Pulmozyme once daily chronically for at least 6 months will continue to receive Pulmozyme once daily for a run-in period of 2 weeks using the Pari LC Plus nebulizer. Patients will then be randomized to receive in a crossover design Pulmozyme once daily for two treatment periods of 2 weeks each using either the Pari LC Plus or the eRapid nebulizer. Anticipated time on study treatment is 6 weeks.

Official Title
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A Phase IV Multicenter, Randomized, Open Label, Two-Period, Crossover Study in Patients With Cystic Fibrosis to Evaluate the Comparable Efficacy and Safety of Pulmozyme Delivered by the eRapid Nebulizer System

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: dornase alfa [Pulmozyme®]
* Drug: dornase alfa [Pulmozyme®]


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Male and female patients, >/= 6 years of age Confirmed diagnosis of cystic fibrosis (CF) Receiving Pulmozyme once daily chronically for treatment of CF for at least 6 months prior to screening Percent predicted FEV1 >/= 40% at screening based on the Wang (males < 18 years, females < 16 years) or Hankinson (males >/= 18 years, females >/= 16 years) standardized equations Able to reproducibly perform spirometry testing and comply with study assessments Exclusion Criteria: An acute respiratory infection or pulmonary exacerbation within 4 weeks prior to randomization Initiation of any new chronic therapy (e.g. inhaled corticosteroids, inhaled oral antibiotics, high-dose ibuprofen, hypertonic saline, ivacaftor) for respiratory disease within 4 weeks prior to randomization Changes in chest physiotherapy schedule within 4 weeks prior to randomization Hospitalization within 4 weeks prior to randomization Planned hospitalization during the 6-week study History of organ transplantation Participation in an investigational drug or device study within 30 day prior to screening

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: eRapid Nebulizer<br>Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks. | Drug: dornase alfa [Pulmozyme®]<br>* Inhaled once daily by Pari eRapid nebulizer.<br>|
| Active Comparator: Jet Nebulizer<br>Dornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks. | Drug: dornase alfa [Pulmozyme®]<br>* Inhaled once daily by Pari LC Plus jet nebulizer.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) | Spirometry was performed according to American Thoracic Society standards. FEV1 is the amount of air that is forced out of the lungs in one second and was measured at the end of each 2-week treatment period. The percent predicted FEV1 was calculated as: Percent predicted FEV1 =FEV1 (L) / Predicted FEV1 (L) ×100. | At the end of each 2-week treatment period | 
| Safety: Number of Participants With Adverse Events During Each Treatment Period | An adverse event was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events. | 4 Weeks |