Back to resultsA Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis
Primary Purpose
Cystic Fibrosis
Status
Terminated
Phase
Phase 4
Locations
Study Type
Interventional
Intervention
Dornase alfa
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis focused on measuring Pulmozyme, CF
Eligibility 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
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Dornase alfa
Placebo
Arm Description
Outcomes
Primary Outcome Measures
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).
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
Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10)
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.
Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10)
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).
Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers
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
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT00680316
Brief Title
A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis
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 Type
Interventional
2. Study Status
Record Verification Date
November 2016
Overall Recruitment Status
Terminated
Why Stopped
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
Study Start Date
June 2008 (undefined)
Primary Completion Date
May 2009 (Actual)
Study Completion Date
May 2009 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Genentech, Inc.
4. Oversight
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
Keywords
Pulmozyme, CF
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
3 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Dornase alfa
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Dornase alfa
Other Intervention Name(s)
Pulmozyme
Intervention Description
2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days
Primary Outcome Measure Information:
Title
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).
Description
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.
Time Frame
from Visit 2 to Visit 3 (16 +/- 2 days)
Secondary Outcome Measure Information:
Title
Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10)
Description
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.
Time Frame
from Visit 2 to Visit 3 (16 +/- 2 days)
Title
Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10)
Description
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).
Time Frame
from Visit 2 to Visit 3 (16 +/- 2 days)
Title
Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers
Description
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
Time Frame
from Visit 2 to Visit 3 (16 +/- 2 days)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Michelle Freemer, M.D.
Organizational Affiliation
Genentech, Inc.
Official's Role
Study Director
12. IPD Sharing Statement
Learn more about this trial
A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis
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