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:
Information provided by:
Genentech
ClinicalTrials.gov Identifier:
NCT00680316
First received: May 16, 2008
Last updated: May 18, 2011
Last verified: May 2011

May 16, 2008
May 18, 2011
June 2008
May 2009   (final data collection date for primary outcome measure)
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.
Change in pulmonary function as measured by oscilllometry [ Time Frame: From Visit 2 to Visit 3 ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00680316 on ClinicalTrials.gov Archive Site
  • 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
  • Change in pulmonary function as measured by oscillometry and spirometry [ Time Frame: From Vist 2 to Visit 3 ] [ Designated as safety issue: No ]
  • Change in quality of life and disease specific symptoms [ Time Frame: From Vist 2 to Visit 3 ] [ Designated as safety issue: No ]
Not Provided
Not Provided
 
A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis
A Phase IV, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme® in 3- to 5-Year-Old Patients With Cystic Fibrosis

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.

Not Provided
Interventional
Phase 4
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Cystic Fibrosis
  • Drug: Dornase alfa
    2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days
    Other Name: Pulmozyme
  • Drug: Placebo
    2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days
  • Experimental: Dornase alfa
    Intervention: Drug: Dornase alfa
  • Placebo Comparator: Placebo
    Intervention: Drug: Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
3
May 2009
May 2009   (final data collection date for primary outcome measure)

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
Both
3 Years to 5 Years
No
Contact information is only displayed when the study is recruiting subjects
Not Provided
 
NCT00680316
Z4240g
Not Provided
Disclosures Group, Genentech, Inc.
Genentech
Not Provided
Study Director: Michelle Freemer, M.D. Genentech
Genentech
May 2011

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP