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This phase IV, multicenter, randomized, open-label, two-period crossover study will evaluate the comparable efficacy and safety of Pulmozyme (dornase alfa) delivered by the eRapid nebulizer system in patients with cystic fibrosis. Patients who have been receiving Pulmozyme once daily chronically for at least 6 months will continue to receive Pulmozyme once daily for a run-in period of 2 weeks using the Pari LC Plus nebulizer. Patients will then be randomized to receive in a crossover design Pulmozyme once daily for two treatment periods of 2 weeks each using either the Pari LC Plus or the eRapid nebulizer. Anticipated time on study treatment is 6 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| eRapid Nebulizer | Experimental | Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks. |
|
| Jet Nebulizer | Active Comparator | Dornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| dornase alfa [Pulmozyme®] | Drug | Inhaled once daily by Pari eRapid nebulizer. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) | 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. | At the end of each 2-week treatment period |
| Safety: Number of Participants With Adverse Events During Each Treatment Period | 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. | 4 Weeks |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Clinical Trials | Genentech, Inc. | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Phoenix | Arizona | 85016 | United States | |||
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33735508 | Derived | Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5. | |
| 25921451 | Derived | Sawicki GS, Chou W, Raimundo K, Trzaskoma B, Konstan MW. Randomized trial of efficacy and safety of dornase alfa delivered by eRapid nebulizer in cystic fibrosis patients. J Cyst Fibros. 2015 Nov;14(6):777-83. doi: 10.1016/j.jcf.2015.04.003. Epub 2015 Apr 25. |
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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.
99 patients were enrolled. 96 unique patients entered the run-in period including 3 patients who entered the run-in period twice.
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| ID | Title | Description |
|---|---|---|
| FG000 | 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. . |
| FG001 | Jet Nebulizer Then eRapid Nebulizer |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Treatment Period 1 |
|
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| dornase alfa [Pulmozyme®] |
| Drug |
Inhaled once daily by Pari LC Plus jet nebulizer. |
|
| Little Rock |
| Arkansas |
| 72202 |
| United States |
| Portland | Maine | 4102 | United States |
| Boston | Massachusetts | 02115 | United States |
| Lebanon | New Hampshire | 03756 | United States |
| Long Branch | New Jersey | 07740 | United States |
| Rochester | New York | 14618 | United States |
| Durham | North Carolina | 27710 | United States |
| Akron | Ohio | 44308 | United States |
| Cleveland | Ohio | 44106 | United States |
| Charleston | South Carolina | 29425 | United States |
| Sioux Falls | South Dakota | 57104 | United States |
| Nashville | Tennessee | 37232-9119 | United States |
| Houston | Texas | 77030 | United States |
| Salt Lake City | Utah | 84132 | United States |
| Seattle | Washington | 98105 | United States |
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. |
| Received Treatment |
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| COMPLETED |
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| NOT COMPLETED |
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| Treatment Period 2 |
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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.
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| ID | Title | Description |
|---|---|---|
| BG000 | 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. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) | 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. | Modified Intent-to-Treat (mITT) population included all randomized participants with baseline and endpoint FEV1 values for both treatment periods. | Posted | Mean | Standard Deviation | percent predicted | At the end of each 2-week treatment period |
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| Primary | Safety: Number of Participants With Adverse Events During Each Treatment Period | 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. | Safety population included all randomized participants who received treatment. | Posted | Number | participants | 4 Weeks |
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Safety population included all randomized participants who received treatment.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | eRapid Nebulizer | Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks. | 0 | 85 | 0 | 85 | ||
| EG001 | Jet Nebulizer | Pulmozyme® inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks. | 0 | 85 | 0 | 85 |
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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.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Medical Communications | Hoffman-LaRoche | 800-821-8590 | genentech@druginfo.com |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
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| ID | Term |
|---|---|
| C568813 | dornase alfa |
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