Cystic FibrosisPhase 4NCT01712334
A Study of the Comparable Efficacy and Safety of Pulmozyme (Dornase Alfa) Delivered by the eRapid Nebulizer System in Patients With Cystic Fibrosis
A Phase 4 study testing dornase alfa [Pulmozyme®] in people with Cystic Fibrosis, run by Genentech, Inc. at 16 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- dornase alfa [Pulmozyme®]
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 99 participants
- Where
- 16 sitesUnited States
- Study dates
- Dec 2012to Jun 2013
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male and female patients, >/= 6 years of age
- Confirmed diagnosis of cystic fibrosis (CF)
- Receiving Pulmozyme once daily chronically for treatment of CF for at least 6 months prior to screening
- Percent predicted FEV1 >/= 40% at screening based on the Wang (males < 18 years, females < 16 years) or Hankinson (males >/= 18 years, females >/= 16 years) standardized equations
- Able to reproducibly perform spirometry testing and comply with study assessments
Who cannot join
- An acute respiratory infection or pulmonary exacerbation within 4 weeks prior to randomization
- Initiation of any new chronic therapy (e.g. inhaled corticosteroids, inhaled oral antibiotics, high-dose ibuprofen, hypertonic saline, ivacaftor) for respiratory disease within 4 weeks prior to randomization
- Changes in chest physiotherapy schedule within 4 weeks prior to randomization
- Hospitalization within 4 weeks prior to randomization
- Planned hospitalization during the 6-week study
- History of organ transplantation
- Participation in an investigational drug or device study within 30 day prior to screening
Trial locations and contacts
16 sites in 1 country.
16 sites in 1 country
United States16 sites
Study site
Akron, Ohio, 44308
Study site
Boston, Massachusetts, 02115
Study site
Charleston, South Carolina, 29425
Study site
Cleveland, Ohio, 44106
Study site
Durham, North Carolina, 27710
Study site
Houston, Texas, 77030
Study site
Lebanon, New Hampshire, 03756
Study site
Little Rock, Arkansas, 72202
Study site
Long Branch, New Jersey, 07740
Study site
Nashville, Tennessee, 37232-9119
Study site
Phoenix, Arizona, 85016
Study site
Portland, Maine, 4102
Study site
Rochester, New York, 14618
Study site
Salt Lake City, Utah, 84132
Study site
Seattle, Washington, 98105
Study site
Sioux Falls, South Dakota, 57104
About this study
Official title: A Phase IV Multicenter, Randomized, Open Label, Two-Period, Crossover Study in Patients With Cystic Fibrosis to Evaluate the Comparable Efficacy and Safety of Pulmozyme Delivered by the eRapid Nebulizer System
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.
- Conditions
- Cystic Fibrosis
- Being tested
- dornase alfa [Pulmozyme®]
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
dornase alfa [Pulmozyme®]Drug
Inhaled once daily by Pari eRapid nebulizer.
dornase alfa [Pulmozyme®]Drug
Inhaled once daily by Pari LC Plus jet nebulizer.
| Study group | Role | What they receive |
|---|---|---|
| eRapid Nebulizer | Experimental | Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks. Receives: Drug: dornase alfa [Pulmozyme®] |
| Jet Nebulizer | Active comparator | Dornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks. Receives: Drug: dornase alfa [Pulmozyme®] |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 4 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2012 (estimated)
- Study startDec 2012
- Primary completionJun 2013
- Study completionJun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Genentech, Inc.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 99 participants (actual)
Sponsor and investigators
- Lead sponsor
- Genentech, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Genentech, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01712334
- Sponsor's protocol ID
- ML28249
- Status last verified
- Mar 2014
- First submitted
- 19 Oct 2012
- Last updated
- 1 May 2014
- Results posted
- Yes, on ClinicalTrials.gov
Publications
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.
PubMed 33735508Sawicki 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.
PubMed 25921451
Study at a glance
From the registry record, last updated by the sponsor on 1 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase IV Multicenter, Randomized, Open Label, Two-Period, Crossover Study in Patients With Cystic Fibrosis to Evaluate the Comparable Efficacy and Safety of Pulmozyme Delivered by the eRapid Nebulizer System
- ClinicalTrials.gov ID
- NCT01712334
- Acronym / sponsor ID
- ML28249
- Conditions
- Cystic Fibrosis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 99 participants (actual)
- Lead sponsor
- Genentech, Inc.Industry (pharma, biotech, devices)
- Study start
- Dec 2012
- Primary completion
- Jun 2013 (actual)
- Study completion
- Jun 2013 (actual)
- First posted
- 23 Oct 2012 (estimated)
- Results
- Posted 1 May 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| eRapid Nebulizer | Experimental | Drug: dornase alfa [Pulmozyme®]Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks. |
| Jet Nebulizer | Active comparator | Drug: dornase alfa [Pulmozyme®]Dornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Male and female patients, >/= 6 years of age
- Confirmed diagnosis of cystic fibrosis (CF)
- Receiving Pulmozyme once daily chronically for treatment of CF for at least 6 months prior to screening
- Percent predicted FEV1 >/= 40% at screening based on the Wang (males < 18 years, females < 16 years) or Hankinson (males >/= 18 years, females >/= 16 years) standardized equations
- Able to reproducibly perform spirometry testing and comply with study assessments
Key exclusion criteria (7 listed)
- An acute respiratory infection or pulmonary exacerbation within 4 weeks prior to randomization
- Initiation of any new chronic therapy (e.g. inhaled corticosteroids, inhaled oral antibiotics, high-dose ibuprofen, hypertonic saline, ivacaftor) for respiratory disease within 4 weeks prior to randomization
- Changes in chest physiotherapy schedule within 4 weeks prior to randomization
- Hospitalization within 4 weeks prior to randomization
- Planned hospitalization during the 6-week study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| dornase alfa [Pulmozyme®]Inhaled once daily by Pari eRapid nebulizer. | Drug | eRapid Nebulizer |
| dornase alfa [Pulmozyme®]Inhaled once daily by Pari LC Plus jet nebulizer. | Drug | Jet Nebulizer |
Primary outcome
Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)
Time frame: At the end of each 2-week treatment period
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.
Safety: Number of Participants With Adverse Events During Each Treatment Period
Time frame: 4 Weeks
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 May 2014.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33735508Yang C, Montgomery M. Dornase alfa for cystic fibrosis. Cochrane Database Syst Rev. 2021 Mar 18;3(3):CD001127. doi: 10.1002/14651858.CD001127.pub5.
- Derived from this study: PubMed 25921451Sawicki 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Genentech, Inc.
16 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing dornase alfa [Pulmozyme®] in people with Cystic Fibrosis, run by Genentech, Inc. at 16 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 1 May 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 May 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) (time frame: At the end of each 2-week treatment period). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01712334, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 May 2014.