Cystic FibrosisPhase 3NCT00534079
Nasal Inhalation of Pulmozyme in Patients With Cystic Fibrosis and Chronic Rhinosinusitis
A Phase 3 study testing Dornase alfa (Pulmozyme) and isotonic saline in people with Cystic Fibrosis and Rhinosinusitis, run by University of Jena at 2 sites in Germany.
Trial summary
Rhinosinusitis disorders are often associated with Cystic Fibrosis. They can restrict quality of life enormously and give cause to repeated ENT surgery. The basic defect in CF is a dysfunction of chloride channels in exocrine glands, leading to retention of secretions and consecutive chronic inflammation with… Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Cystic Fibrosis, Rhinosinusitis
- What is being tested?
- Dornase alfa (Pulmozyme) and isotonic saline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 23 participants
- Where
- 2 sitesGermany
- Study dates
- Sep 2007to Feb 2009
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject has a confirmed diagnosis of cystic fibrosis based on: 3 positive sweat chloride tests and/or genetic characterisation
- Subject is 5 years of age or older
- Subject has chronic or recurrent rhinosinusitic disorders
- Subject is able to comply with the procedures scheduled in the protocol
- Women of childbearing potential are only included into the study, if they are using an effective method of birth control during the protocol (e.g. implants, combined oral contraceptives, injectables, some IUDs, sexual abstinence or vasectomised partner)
Who cannot join
- Subject has a critical condition defined as: FEV1 < 30% and / or SaO2 < 93% without O2-substitution; need of O2-substitution
- Subject had an ENT surgery within 6 months prior to study
- Subject shows signs of nasal bleeding
- Subject has an ear drum perforation
- Subject had an acute rhinosinusitis or a pulmonary exacerbation at study entry with need of additional systemic antibiotic therapy
- Subject has a new therapy with nasal topic steroids during treatment interval
Show all 11Show fewer
- Subject has a new systemic steroid therapy
- Subject is unlikely to comply with the procedures scheduled in the protocol
- Subject has a known allergic reaction to the medication
- Subject is pregnant or breastfeeding
- Patient participates in another clinical trial within 30 days prior to study entry or 30 days after end of the study.
Trial locations and contacts
2 sites in 1 country.
Germany2 sites
Mukoviszidosezentrum der Friedrich-Schiller-Universität
Jena, Thuringia, 07740
Universitäts-Kinderklinik
Tübingen, Baden-Würtemberg, 72076
About this study
Official title: Nasal Inhalation of Dornase Alfa (Pulmozyme) in Patients With Cystic Fibrosis and Chronic Rhinosinusitis - a Double Blind Placebo-controlled Cross-over, Bicenter, Prospective Clinical Study
Rhinosinusitis disorders are often associated with Cystic Fibrosis. They can restrict quality of life enormously and give cause to repeated ENT surgery. The basic defect in CF is a dysfunction of chloride channels in exocrine glands, leading to retention of secretions and consecutive chronic inflammation with bacterial superinfections.
The prospective placebo controlled cross-over study aims at the evaluation of a nasally inhalation of Pulmozyme with respect to mucus retention and resulting inflammation.
- Conditions
- Cystic Fibrosis, Rhinosinusitis
- Being tested
- Dornase alfa (Pulmozyme), isotonic saline
- Keywords
- Cystic Fibrosis and chronic rhinosinusitis
- Medical subject headings
- Cystic Fibrosis, Rhinosinusitis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Dornase alfa (Pulmozyme)Drug
1 x 2,5 ml per day, inhalation use, for 28 days
isotonic salineDrug
1 x 2,5 ml per day, inhalation use, for 28 days
| Study group | Role | What they receive |
|---|---|---|
| Dornase alfa | Experimental | 28 days of sinonasal inhalation (Pari Sinus) Receives: Drug: Dornase alfa (Pulmozyme); Drug: isotonic saline |
| isotonic saline | Placebo comparator | 28 days of sinonasal inhalation (Pari Sinus) Receives: Drug: Dornase alfa (Pulmozyme); Drug: isotonic saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Variations in the Sino-nasal-outcome test SNOT-20 adapt CF (primary nasal parameters: obstruction of nasal breathing, sneeze stimulus, permanent nose running, thick-mucous nasal discharge, earaches)
Measured: day 1, 29, 57 and 85
Secondary outcomes (7)
Variations in the Sino-nasal-outcome test SNOT-20 adapt CF (secondary nasal parameters, general quality of live parameters and total SNOT 20 adapt. CF score)
Measured: day 1, 29, 57 and 85
Changes of pathological alterations visible in MRT images of nose and paranasal sinuses (in selected patients)
Measured: day 1, 29, 57, 85
Changes in the nasal lavage fluid and in the serological markers of inflammation
Measured: day 1, 29, 57 and 85
Changes in rhinoscopic findings
Measured: day 1, 29, 57 and 85
Changes in rhinomanometric findings
Measured: day 1, 29, 27 and 85
Incidence of rhinosinusitic and pulmonary exacerbations during therapy
Measured: day 1 - 85
Need for decongestants or nasal lavage during treatment
Measured: day 1 - 85
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Sep 2007 (estimated)
- Study startSep 2007
- Primary completionFeb 2009
- Study completionFeb 2009
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Jena
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, PD Dr. Jochen G. Mainz, University of Jena
- Investigators
- Jochen Mainz, M.D., Study chair, University of Jena
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00534079
- Sponsor's protocol ID
- pulmozyme-nasal-cf
- Status last verified
- Dec 2014
- First submitted
- 21 Sep 2007
- Last updated
- 4 Dec 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Mainz JG, Michl R, Arnold C. Response to Karadag. J Cyst Fibros. 2014 Sep;13(5):602-3. doi: 10.1016/j.jcf.2014.07.005. Epub 2014 Jul 28. No abstract available.
PubMed 25081561Mainz JG, Schien C, Schiller I, Schadlich K, Koitschev A, Koitschev C, Riethmuller J, Graepler-Mainka U, Wiedemann B, Beck JF. Sinonasal inhalation of dornase alfa administered by vibrating aerosol to cystic fibrosis patients: a double-blind placebo-controlled cross-over trial. J Cyst Fibros. 2014 Jul;13(4):461-70. doi: 10.1016/j.jcf.2014.02.005. Epub 2014 Mar 1.
PubMed 24594542
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nasal Inhalation of Dornase Alfa (Pulmozyme) in Patients With Cystic Fibrosis and Chronic Rhinosinusitis - a Double Blind Placebo-controlled Cross-over, Bicenter, Prospective Clinical Study
- ClinicalTrials.gov ID
- NCT00534079
- Acronym / sponsor ID
- pulmozyme-nasal-cf
- Conditions
- Cystic Fibrosis, Rhinosinusitis
- Keywords
- Cystic Fibrosis and chronic rhinosinusitis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- University of JenaUniversity, hospital or other
- Study start
- Sep 2007
- Primary completion
- Feb 2009 (actual)
- Study completion
- Feb 2009 (actual)
- First posted
- 24 Sep 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Rhinosinusitis disorders are often associated with Cystic Fibrosis. They can restrict quality of life enormously and give cause to repeated ENT surgery. The basic defect in CF is a dysfunction of chloride channels in exocrine glands, leading to retention of secretions and consecutive chronic inflammation with bacterial superinfections.
The prospective placebo controlled cross-over study aims at the evaluation of a nasally inhalation of Pulmozyme with respect to mucus retention and resulting inflammation.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Dornase alfa | Experimental | Drug: Dornase alfa (Pulmozyme), Drug: isotonic saline28 days of sinonasal inhalation (Pari Sinus) |
| isotonic saline | Placebo comparator | Drug: Dornase alfa (Pulmozyme), Drug: isotonic saline28 days of sinonasal inhalation (Pari Sinus) |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Subject has a confirmed diagnosis of cystic fibrosis based on: 3 positive sweat chloride tests and/or genetic characterisation
- Subject is 5 years of age or older
- Subject has chronic or recurrent rhinosinusitic disorders
- Subject is able to comply with the procedures scheduled in the protocol
- Women of childbearing potential are only included into the study, if they are using an effective method of birth control during the protocol (e.g. implants, combined oral contraceptives, injectables, some IUDs, sexual abstinence or vasectomised partner)
Key exclusion criteria (11 listed)
- Subject has a critical condition defined as: FEV1 < 30% and / or SaO2 < 93% without O2-substitution; need of O2-substitution
- Subject had an ENT surgery within 6 months prior to study
- Subject shows signs of nasal bleeding
- Subject has an ear drum perforation
- Subject had an acute rhinosinusitis or a pulmonary exacerbation at study entry with need of additional systemic antibiotic therapy
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)1 x 2,5 ml per day, inhalation use, for 28 days | Drug | Dornase alfa, isotonic saline |
| isotonic saline1 x 2,5 ml per day, inhalation use, for 28 days | Drug | Dornase alfa, isotonic saline |
Primary outcome
Variations in the Sino-nasal-outcome test SNOT-20 adapt CF (primary nasal parameters: obstruction of nasal breathing, sneeze stimulus, permanent nose running, thick-mucous nasal discharge, earaches)
Time frame: day 1, 29, 57 and 85
7 secondary outcomes
Variations in the Sino-nasal-outcome test SNOT-20 adapt CF (secondary nasal parameters, general quality of live parameters and total SNOT 20 adapt. CF score)
Time frame: day 1, 29, 57 and 85
Changes of pathological alterations visible in MRT images of nose and paranasal sinuses (in selected patients)
Time frame: day 1, 29, 57, 85
Changes in the nasal lavage fluid and in the serological markers of inflammation
Time frame: day 1, 29, 57 and 85
Changes in rhinoscopic findings
Time frame: day 1, 29, 57 and 85
Changes in rhinomanometric findings
Time frame: day 1, 29, 27 and 85
Incidence of rhinosinusitic and pulmonary exacerbations during therapy
Time frame: day 1 - 85
Need for decongestants or nasal lavage during treatment
Time frame: day 1 - 85
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2014.
- Background: PubMed 25081561Mainz JG, Michl R, Arnold C. Response to Karadag. J Cyst Fibros. 2014 Sep;13(5):602-3. doi: 10.1016/j.jcf.2014.07.005. Epub 2014 Jul 28. No abstract available.
- Results publication: PubMed 24594542Mainz JG, Schien C, Schiller I, Schadlich K, Koitschev A, Koitschev C, Riethmuller J, Graepler-Mainka U, Wiedemann B, Beck JF. Sinonasal inhalation of dornase alfa administered by vibrating aerosol to cystic fibrosis patients: a double-blind placebo-controlled cross-over trial. J Cyst Fibros. 2014 Jul;13(4):461-70. doi: 10.1016/j.jcf.2014.02.005. Epub 2014 Mar 1.
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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.
Jochen Mainz, M.D. study chair
University of Jena
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Dornase alfa (Pulmozyme) and isotonic saline in people with Cystic Fibrosis and Rhinosinusitis, run by University of Jena at 2 sites in Germany.
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 11 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 4 Dec 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Germany. 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Variations in the Sino-nasal-outcome test SNOT-20 adapt CF (primary nasal parameters: obstruction of nasal breathing, sneeze stimulus, permanent nose running, thick-mucous nasal discharge, earaches) (time frame: day 1, 29, 57 and 85). 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 NCT00534079, 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 4 Dec 2014.