A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis
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
1 other identifier
interventional
99
1 country
16
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_4
Started Dec 2012
Shorter than P25 for phase_4
16 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 19, 2012
CompletedFirst Posted
Study publicly available on registry
October 23, 2012
CompletedStudy Start
First participant enrolled
December 1, 2012
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 1, 2013
CompletedStudy Completion
Last participant's last visit for all outcomes
June 1, 2013
CompletedResults Posted
Study results publicly available
May 1, 2014
CompletedMay 1, 2014
March 1, 2014
6 months
October 19, 2012
March 27, 2014
March 31, 2014
Conditions
Outcome Measures
Primary Outcomes (2)
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
Study Arms (2)
eRapid Nebulizer
EXPERIMENTALDornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.
Jet Nebulizer
ACTIVE COMPARATORDornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.
Interventions
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Genentech, Inc.lead
Study Sites (16)
Unknown Facility
Phoenix, Arizona, 85016, United States
Unknown Facility
Little Rock, Arkansas, 72202, United States
Unknown Facility
Portland, Maine, 4102, United States
Unknown Facility
Boston, Massachusetts, 02115, United States
Unknown Facility
Lebanon, New Hampshire, 03756, United States
Unknown Facility
Long Branch, New Jersey, 07740, United States
Unknown Facility
Rochester, New York, 14618, United States
Unknown Facility
Durham, North Carolina, 27710, United States
Unknown Facility
Akron, Ohio, 44308, United States
Unknown Facility
Cleveland, Ohio, 44106, United States
Unknown Facility
Charleston, South Carolina, 29425, United States
Unknown Facility
Sioux Falls, South Dakota, 57104, United States
Unknown Facility
Nashville, Tennessee, 37232-9119, United States
Unknown Facility
Houston, Texas, 77030, United States
Unknown Facility
Salt Lake City, Utah, 84132, United States
Unknown Facility
Seattle, Washington, 98105, United States
Related Publications (2)
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.
PMID: 33735508DERIVEDSawicki 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.
PMID: 25921451DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Communications
- Organization
- Hoffman-LaRoche
Study Officials
- STUDY DIRECTOR
Clinical Trials
Genentech, Inc.
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- CROSSOVER
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 19, 2012
First Posted
October 23, 2012
Study Start
December 1, 2012
Primary Completion
June 1, 2013
Study Completion
June 1, 2013
Last Updated
May 1, 2014
Results First Posted
May 1, 2014
Record last verified: 2014-03