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

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01712334
• Other study ID #: ML28249
• Status: Completed
• Phase: Phase 4
• First received: October 19, 2012
• Last updated: March 31, 2014
• Start date: December 2012
• Est. completion date: June 2013

Study information

• Verified date: March 2014
• Source: Genentech, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 99
• Est. completion date: June 2013
• Est. primary completion date: June 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years and older

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• dornase alfa [Pulmozyme®]
Inhaled once daily by Pari eRapid nebulizer.
• dornase alfa [Pulmozyme®]
Inhaled once daily by Pari LC Plus jet nebulizer.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Genentech, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	No
Primary	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	No