Clinical trial NCT00534079: Nasal Inhalation of Pulmozyme in Patients With Cystic Fibrosis and Chronic Rhinosinusitis
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Rhinosinusitis
Keywords: Cystic Fibrosis and chronic rhinosinusitis
Lead sponsor: University of Jena
Sponsor class: OTHER
First posted: 2007-09-24
Enrollment: 23
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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 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.


Eligibility criteria: Inclusion Criteria:

* 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)

Exclusion Criteria:

* 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
* 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.

Primary outcomes:
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)
timeFrame: day 1, 29, 57 and 85
Secondary outcomes:
measure: 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)
timeFrame: day 1, 29, 57 and 85
measure: Changes of pathological alterations visible in MRT images of nose and paranasal sinuses (in selected patients)
timeFrame: day 1, 29, 57, 85
measure: Changes in the nasal lavage fluid and in the serological markers of inflammation
timeFrame: day 1, 29, 57 and 85
measure: Changes in rhinoscopic findings
timeFrame: day 1, 29, 57 and 85
measure: Changes in rhinomanometric findings
timeFrame: day 1, 29, 27 and 85
measure: Incidence of rhinosinusitic and pulmonary exacerbations during therapy
timeFrame: day 1 - 85
measure: Need for decongestants or nasal lavage during treatment
timeFrame: day 1 - 85
Other outcomes:
Locations:
city: Tübingen
country: Germany
facility: Universitäts-Kinderklinik
state: Baden-Würtemberg
city: Jena
country: Germany
facility: Mukoviszidosezentrum der Friedrich-Schiller-Universität
state: Thüringen
Study officials:
affiliation: University of Jena
name: Jochen Mainz, M.D.
role: STUDY_CHAIR
References:
citation: 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.
citation: Mainz 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.