We updated the design of this site on December 18, 2017. Learn more.
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT00680316
Recruitment Status : Terminated (Most patients were unable to perform the test being used to measure lung function, thus it became clear the study would not yield clinically meaningful data)
First Posted : May 20, 2008
Results First Posted : June 17, 2011
Last Update Posted : January 12, 2017
Sponsor:
Information provided by (Responsible Party):

Study Description
Brief Summary:
This was a Phase IV, multicenter, randomized, double-blind, placebo-controlled trial designed to evaluate the effect of Pulmozyme on pulmonary function, health-related quality of life (HRQOL), and respiratory symptoms in 3- to 5-year-old children with cystic fibrosis (CF). Approximately 40 patients were planned to be enrolled in this study. However, only 3 patients were eligible for random allocation and received treatment: 1 patient in the Pulmozyme group and 2 patients in the placebo group. All 3 patients completed the study assessments but did not have usable pulmonary function test (PFT) data.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: Dornase alfa Drug: Placebo Phase 4

Study Design

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase IV, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme® in 3- to 5-Year-Old Patients With Cystic Fibrosis
Study Start Date : June 2008
Primary Completion Date : May 2009
Study Completion Date : May 2009

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
U.S. FDA Resources

Arms and Interventions

Arm Intervention/treatment
Experimental: Dornase alfa Drug: Dornase alfa
2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days
Other Name: Pulmozyme
Placebo Comparator: Placebo Drug: Placebo
2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days


Outcome Measures

Primary Outcome Measures :
  1. Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment). [ Time Frame: from Visit 2 to Visit 3 (16 +/- 2 days) ]
    The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.


Secondary Outcome Measures :
  1. Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10) [ Time Frame: from Visit 2 to Visit 3 (16 +/- 2 days) ]
    The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.

  2. Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10) [ Time Frame: from Visit 2 to Visit 3 (16 +/- 2 days) ]
    The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Resistance is complex measure that incorporates the lack of changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (10Hz was used for the secondary endpoint).

  3. Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers [ Time Frame: from Visit 2 to Visit 3 (16 +/- 2 days) ]

    The CFQ-R for Preschoolers and the CFQ-R for Parents of Preschoolers was designed specifically to measure the impact of CF for patients with a diagnosis of CF. Each question is answered using a 4-point Likert scale.

    In order to calculate the domain/symptom scale scores, the following algorithm is followed

    • Re-number items which have been reverse coded
    • Calculate the mean of the items to be included. If more than half of the items are missing, then the score is considered missing
    • Re-scale to result in a scaled score which ranges from 0 to 100, with higher scores indicating better health


Eligibility Criteria

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   3 Years to 5 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Signed Informed Consent Form
  • Aged 3-5 years
  • Diagnosis of cystic fibrosis

Exclusion Criteria:

  • Children taking scheduled inhaled Pulmozyme or hypertonic saline within 56 days prior to Visit 1 or any Pulmozyme in the 28 days before Visit 1
  • Involvement in a clinical intervention trial within the 4 weeks prior to Visit 1
  • Use of an investigational drug or device within 28 days prior to Visit 1
  • Any other condition that might increase the risk of participation to the patient in the judgement of the investigator
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00680316


Sponsors and Collaborators
Genentech, Inc.
Investigators
Study Director: Michelle Freemer, M.D. Genentech, Inc.
More Information

Responsible Party: Genentech, Inc.
ClinicalTrials.gov Identifier: NCT00680316     History of Changes
Other Study ID Numbers: Z4240g
First Posted: May 20, 2008    Key Record Dates
Results First Posted: June 17, 2011
Last Update Posted: January 12, 2017
Last Verified: November 2016

Keywords provided by Genentech, Inc.:
Pulmozyme
CF

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