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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

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
dornase alfa [Pulmozyme®]
dornase alfa [Pulmozyme®]
Sponsored by
Genentech, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

eRapid Nebulizer

Jet Nebulizer

Arm Description

Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.

Dornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.

Outcomes

Primary Outcome Measures

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.
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.

Secondary Outcome Measures

Full Information

First Posted
October 19, 2012
Last Updated
March 31, 2014
Sponsor
Genentech, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT01712334
Brief Title
A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis
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
Study Type
Interventional

2. Study Status

Record Verification Date
March 2014
Overall Recruitment Status
Completed
Study Start Date
December 2012 (undefined)
Primary Completion Date
June 2013 (Actual)
Study Completion Date
June 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genentech, Inc.

4. Oversight

5. Study Description

Brief 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
99 (Actual)

8. Arms, Groups, and Interventions

Arm Title
eRapid Nebulizer
Arm Type
Experimental
Arm Description
Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.
Arm Title
Jet Nebulizer
Arm Type
Active Comparator
Arm Description
Dornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.
Intervention Type
Drug
Intervention Name(s)
dornase alfa [Pulmozyme®]
Intervention Description
Inhaled once daily by Pari eRapid nebulizer.
Intervention Type
Drug
Intervention Name(s)
dornase alfa [Pulmozyme®]
Intervention Description
Inhaled once daily by Pari LC Plus jet nebulizer.
Primary Outcome Measure Information:
Title
Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)
Description
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.
Time Frame
At the end of each 2-week treatment period
Title
Safety: Number of Participants With Adverse Events During Each Treatment Period
Description
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.
Time Frame
4 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trials
Organizational Affiliation
Genentech, Inc.
Official's Role
Study Director
Facility Information:
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
City
Portland
State/Province
Maine
ZIP/Postal Code
4102
Country
United States
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
City
Lebanon
State/Province
New Hampshire
ZIP/Postal Code
03756
Country
United States
City
Long Branch
State/Province
New Jersey
ZIP/Postal Code
07740
Country
United States
City
Rochester
State/Province
New York
ZIP/Postal Code
14618
Country
United States
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
City
Akron
State/Province
Ohio
ZIP/Postal Code
44308
Country
United States
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
City
Sioux Falls
State/Province
South Dakota
ZIP/Postal Code
57104
Country
United States
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232-9119
Country
United States
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
City
Seattle
State/Province
Washington
ZIP/Postal Code
98105
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
33735508
Citation
Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5.
Results Reference
derived
PubMed Identifier
25921451
Citation
Sawicki GS, Chou W, Raimundo K, Trzaskoma B, Konstan MW. Randomized trial of efficacy and safety of dornase alfa delivered by eRapid nebulizer in cystic fibrosis patients. J Cyst Fibros. 2015 Nov;14(6):777-83. doi: 10.1016/j.jcf.2015.04.003. Epub 2015 Apr 25.
Results Reference
derived

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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

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