A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Genentech
ClinicalTrials.gov Identifier:
NCT01712334
First received: October 19, 2012
Last updated: March 31, 2014
Last verified: March 2014
Results First Received: March 27, 2014  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Crossover Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Cystic Fibrosis
Intervention: Drug: dornase alfa [Pulmozyme®]

  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
99 patients were enrolled. 96 unique patients entered the run-in period including 3 patients who entered the run-in period twice.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Patients received dornase alfa (Pulmozyme®) by LC Plus nebulizer in the 2-week run-in period prior to randomization. A total of 86 unique patients were randomized in the study in 87 randomization events. Of the randomized patients, 85 patients completed the study in two treatment sequences.

Reporting Groups
  Description
eRapid Nebulizer Then Jet Nebulizer

Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks in Treatment Period 1 then Pulmozyme® inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks in Treatment Period 2.

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Jet Nebulizer Then eRapid Nebulizer Pulmozyme® inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks in Treatment Period 1 then Pulmozyme® inhaled once daily by the Pari eRapid nebulizer for 2 weeks in Treatment Period 2.

Participant Flow for 2 periods

Period 1:   Treatment Period 1
    eRapid Nebulizer Then Jet Nebulizer     Jet Nebulizer Then eRapid Nebulizer  
STARTED     44     43  
Received Treatment     44     41  
COMPLETED     44     41  
NOT COMPLETED     0     2  
Randomized in Error                 0                 2  

Period 2:   Treatment Period 2
    eRapid Nebulizer Then Jet Nebulizer     Jet Nebulizer Then eRapid Nebulizer  
STARTED     44     41  
COMPLETED     44     41  
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.
Baseline measures were based on the modified Intent-to-Treat (mITT) population that included all participants randomized with both baseline and endpoint Forced Expiratory Volume in 1 second (FEV1) values for both treatment periods.

Reporting Groups
  Description
All Participants All participants received dornase alfa (Pulmozyme) inhaled once daily by the Pari eRapid nebulizer or the Pari LC Plus jet nebulizer for 2 weeks in Treatment Period 1 then crossed over to use the other nebulizer in Treatment Period 2 for 2 weeks.

Baseline Measures
    All Participants  
Number of Participants  
[units: participants]
  85  
Age  
[units: years]
Mean ± Standard Deviation
  13.6  ± 6.92  
Gender  
[units: participants]
 
Female     43  
Male     42  



  Outcome Measures
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1.  Primary:   Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)   [ Time Frame: At the end of each 2-week treatment period ]

2.  Primary:   Safety: Number of Participants With Adverse Events During Each Treatment Period   [ Time Frame: 4 Weeks ]


  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
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  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: Hoffman-LaRoche
phone: 800-821-8590
e-mail: genentech@druginfo.com


No publications provided


Responsible Party: Genentech
ClinicalTrials.gov Identifier: NCT01712334     History of Changes
Other Study ID Numbers: ML28249
Study First Received: October 19, 2012
Results First Received: March 27, 2014
Last Updated: March 31, 2014
Health Authority: United States: Food and Drug Administration