Trial Outcomes & Findings for A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis (NCT NCT01712334)
NCT ID: NCT01712334
Last Updated: 2014-05-01
Results Overview
Spirometry was performed according to American Thoracic Society standards. FEV1 is the amount of air that is forced out of the lungs in one second and was measured at the end of each 2-week treatment period. The percent predicted FEV1 was calculated as: Percent predicted FEV1 =FEV1 (L) / Predicted FEV1 (L) ×100.
COMPLETED
PHASE4
99 participants
At the end of each 2-week treatment period
2014-05-01
Participant Flow
99 patients were enrolled. 96 unique patients entered the run-in period including 3 patients who entered the run-in period twice.
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.
Participant milestones
| Measure |
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.
.
|
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.
|
|---|---|---|
|
Treatment Period 1
STARTED
|
44
|
43
|
|
Treatment Period 1
Received Treatment
|
44
|
41
|
|
Treatment Period 1
COMPLETED
|
44
|
41
|
|
Treatment Period 1
NOT COMPLETED
|
0
|
2
|
|
Treatment Period 2
STARTED
|
44
|
41
|
|
Treatment Period 2
COMPLETED
|
44
|
41
|
|
Treatment Period 2
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
| Measure |
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.
.
|
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.
|
|---|---|---|
|
Treatment Period 1
Randomized in Error
|
0
|
2
|
Baseline Characteristics
A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis
Baseline characteristics by cohort
| Measure |
All Participants
n=85 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.
|
|---|---|
|
Age, Continuous
|
13.6 years
STANDARD_DEVIATION 6.92 • n=5 Participants
|
|
Sex: Female, Male
Female
|
43 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
42 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: At the end of each 2-week treatment periodPopulation: Modified Intent-to-Treat (mITT) population included all randomized participants with baseline and endpoint FEV1 values for both treatment periods.
Spirometry was performed according to American Thoracic Society standards. FEV1 is the amount of air that is forced out of the lungs in one second and was measured at the end of each 2-week treatment period. The percent predicted FEV1 was calculated as: Percent predicted FEV1 =FEV1 (L) / Predicted FEV1 (L) ×100.
Outcome measures
| Measure |
eRapid Nebulizer
n=85 Participants
Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.
|
Jet Nebulizer
n=85 Participants
Pulmozyme® inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.
|
|---|---|---|
|
Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)
|
98.1 percent predicted
Standard Deviation 22.1
|
97.2 percent predicted
Standard Deviation 20.7
|
PRIMARY outcome
Timeframe: 4 WeeksPopulation: Safety population included all randomized participants who received treatment.
An adverse event was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events.
Outcome measures
| Measure |
eRapid Nebulizer
n=85 Participants
Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.
|
Jet Nebulizer
n=85 Participants
Pulmozyme® inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.
|
|---|---|---|
|
Safety: Number of Participants With Adverse Events During Each Treatment Period
|
18 participants
|
24 participants
|
Adverse Events
eRapid Nebulizer
Jet Nebulizer
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The Study being conducted under this Agreement is part of the Overall Study. Investigator is free to publish in reputable journals or to present at professional conferences the results of the Study, but only after the first publication or presentation that involves the Overall Study. The Sponsor may request that Confidential Information be deleted and/or the publication be postponed in order to protect the Sponsor's intellectual property rights.
- Publication restrictions are in place
Restriction type: OTHER