A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis
This study has been 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)
Sponsor:
Genentech
Information provided by:
Genentech
ClinicalTrials.gov Identifier:
NCT00680316
First received: May 16, 2008
Last updated: May 18, 2011
Last verified: May 2011
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Purpose
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 | Intervention | Phase |
|---|---|---|
|
Cystic Fibrosis |
Drug: Dornase alfa Drug: Placebo |
Phase 4 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Intervention Model: Parallel Assignment Masking: Double Blind (Subject, 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
Drug Information available for:
Dornase alfa
U.S. FDA Resources
Further study details as provided by Genentech:
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). [ Time Frame: from Visit 2 to Visit 3 (16 +/- 2 days) ] [ Designated as safety issue: No ]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) [ Time Frame: from Visit 2 to Visit 3 (16 +/- 2 days) ] [ Designated as safety issue: No ]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) [ Time Frame: from Visit 2 to Visit 3 (16 +/- 2 days) ] [ Designated as safety issue: No ]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 [ Time Frame: from Visit 2 to Visit 3 (16 +/- 2 days) ] [ Designated as safety issue: No ]
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
| Enrollment: | 3 |
| Study Start Date: | June 2008 |
| Study Completion Date: | May 2009 |
| Primary Completion Date: | May 2009 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| 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
|
Eligibility| Ages Eligible for Study: | 3 Years to 5 Years |
| Genders Eligible for Study: | Both |
| 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 More Information
Additional Information:
No publications provided
| Responsible Party: | Disclosures Group, Genentech, Inc. |
| ClinicalTrials.gov Identifier: | NCT00680316 History of Changes |
| Other Study ID Numbers: | Z4240g |
| Study First Received: | May 16, 2008 |
| Results First Received: | August 31, 2010 |
| Last Updated: | May 18, 2011 |
| Health Authority: | United States: Food and Drug Administration |
Keywords provided by Genentech:
|
Pulmozyme CF |
Additional relevant MeSH terms:
|
Cystic Fibrosis Fibrosis Pancreatic Diseases Digestive System Diseases Lung Diseases |
Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Pathologic Processes |
ClinicalTrials.gov processed this record on May 19, 2013