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

Phase 1
Conditions
Cystic Fibrosis
MedDRA version: 19.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Registration Number
EUCTR2015-003537-99-Outside-EU/EEA
Lead Sponsor
F. Hoffmann-La Roche AG
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
A
Sex
All
Target Recruitment
80
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
Are the trial subjects under 18? yes
Number of subjects for this age range: 40
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

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

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: The primary objectives of this study are to compare the following:<br>- Stability of lung function as assessed by FEV1 in patients with cystic fibrosis who are maintained on Pulmozyme® when delivered by eRapid vs Pari LC® Plus jet nebulizer system<br>- Safety as assessed by incidence of adverse events in patients with cystic fibrosis who are maintained on Pulmozyme® when delivered by eRapid vs Pari LC® Plus jet nebulizer system;Secondary Objective: Not applicable;Primary end point(s): - Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) <br>- Safety: Number of Participants With Adverse Events During Each Treatment Period;Timepoint(s) of evaluation of this end point: - Stability of Lung Function: At the end of each 2-week treatment period <br>- Safety: 4 weeks
Secondary Outcome Measures
NameTimeMethod
Secondary end point(s): Not applicable;Timepoint(s) of evaluation of this end point: Not applicable
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