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
Results First Received: August 31, 2010  
Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Treatment
Condition: Cystic Fibrosis
Interventions: Drug: Dornase alfa
Drug: Placebo

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Dornase Alfa 2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days
Placebo 2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days

Participant Flow:   Overall Study
    Dornase Alfa     Placebo  
STARTED     1     2  
COMPLETED     1     2  
NOT COMPLETED     0     0  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Dornase Alfa 2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days
Placebo 2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days
Total Total of all reporting groups

Baseline Measures
    Dornase Alfa     Placebo     Total  
Number of Participants  
[units: participants]
  1     2     3  
Age  
[units: years]
Mean ± Standard Deviation
  5.6     4.6  ± 1.5     4.9  ± 1.2  
Gender  
[units: participants]
     
Female     1     0     1  
Male     0     2     2  



  Outcome Measures
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1.  Primary:   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) ]

2.  Secondary:   Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10)   [ Time Frame: from Visit 2 to Visit 3 (16 +/- 2 days) ]

3.  Secondary:   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) ]

4.  Secondary:   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) ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Children were unable to perform forced oscillometry (FOT) or did not remain stable during the study. No efficacy analyses were performed because no patients had complete (pre- or post-treatment) data for pulmonary function tests, including FOT.


  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Medical Communications
Organization: Genentech, Inc.
phone: 800-821-8590


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