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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Genentech
ClinicalTrials.gov Identifier:
NCT01712334
First received: October 19, 2012
Last updated: March 31, 2014
Last verified: March 2014
  Purpose

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.


Condition Intervention Phase
Cystic Fibrosis
Drug: dornase alfa [Pulmozyme®]
Phase 4

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

Resource links provided by NLM:


Further study details as provided by Genentech:

Primary Outcome Measures:
  • Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) [ Time Frame: At the end of each 2-week treatment period ] [ Designated as safety issue: No ]
    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 [ Time Frame: 4 Weeks ] [ Designated as safety issue: No ]
    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.


Enrollment: 99
Study Start Date: December 2012
Study Completion Date: June 2013
Primary Completion Date: June 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: eRapid Nebulizer
Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.
Drug: dornase alfa [Pulmozyme®]
Inhaled once daily by Pari eRapid nebulizer.
Active Comparator: Jet Nebulizer
Dornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.
Drug: dornase alfa [Pulmozyme®]
Inhaled once daily by Pari LC Plus jet nebulizer.

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01712334

Locations
United States, Arizona
Phoenix, Arizona, United States, 85016
United States, Arkansas
Little Rock, Arkansas, United States, 72202
United States, Maine
Portland, Maine, United States, 4102
United States, Massachusetts
Boston, Massachusetts, United States, 02115
United States, New Hampshire
Lebanon, New Hampshire, United States, 03756
United States, New Jersey
Long Branch, New Jersey, United States, 07740
United States, New York
Rochester, New York, United States, 14618
United States, North Carolina
Durham, North Carolina, United States, 27710
United States, Ohio
Akron, Ohio, United States, 44308
Cleveland, Ohio, United States, 44106
United States, South Carolina
Charleston, South Carolina, United States, 29425
United States, South Dakota
Sioux Falls, South Dakota, United States, 57104
United States, Tennessee
Nashville, Tennessee, United States, 37232-9119
United States, Texas
Houston, Texas, United States, 77030
United States, Utah
Salt Lake City, Utah, United States, 84132
United States, Washington
Seattle, Washington, United States, 98105
Sponsors and Collaborators
Genentech
Investigators
Study Director: Clinical Trials Genentech
  More Information

No publications provided

Responsible Party: Genentech
ClinicalTrials.gov Identifier: NCT01712334     History of Changes
Other Study ID Numbers: ML28249
Study First Received: October 19, 2012
Results First Received: March 27, 2014
Last Updated: March 31, 2014
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on September 22, 2014