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
  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:


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
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: NCT00680316

Sponsors and Collaborators
Genentech
Investigators
Study Director: Michelle Freemer, M.D. Genentech
  More 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 August 28, 2014