Nasally Delivered Pulmozyme for Sinusitis in Cystic Fibrosis

Study Overview
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Brief Summary
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Chronic sinusitis is a frequent complication in cystic fibrosis. The aim of this study is to determine whether Pulmozyme(dornase alfa) would maintain sinus health (compared to placebo) in patients with cystic fibrosis who have recently undergone sinus surgery.

Detailed Description
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AIM: To evaluate the effectiveness of Pulmozyme(dornase alfa) in decreasing post-operative sinusitis symptoms in patients with cystic fibrosis (CF) PROCEDURES: 20 patients with CF will be randomized to receive either Pulmozyme or placebo via nasal inhalation daily for 12 months. Consent will be obtained following surgery and treatment will begin 1 week post-operatively. Monitoring will include examination and recording of adverse effects and follow up weekly for one month and then at 2.5, 6, 9 and 12 months. Outcome measures will include ciliary function testing, pulmonary function testing, sinus questionnaires and CT scan.

Official Title
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The Use of Nasally Delivered Pulmozyme in the Treatment of Sinusitis in Cystic Fibrosis Patients: A Pilot Study

Conditions
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Sinusitis, Cystic Fibrosis

Intervention / Treatment
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* Drug: Pulmozyme (dornase alfa)
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Clinical and laboratory diagnosis of cystic fibrosis Age greater than or equal to 5 years Forced expiratory volume in 1 second (FEV1) greater than or equal to 40% predicted Sinus surgery within one week of enrollment Exclusion Criteria: Pregnancy Intolerance of orally inhaled Pulmozyme (dornase alfa)

Ages Eligible for Study
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Minimum Age: 5 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Pulmozyme<br>2.5 mg Pulmozyme (dornase alfa) delivered intranasally once daily | Drug: Pulmozyme (dornase alfa)<br>* 2.5 mg/2.5 mL of Pulmozyme (dornase alfa) delivered via Sinustar nasal nebulizer device<br>* Other names: Pulmozyme, dornase alfa, human recombinant DNase 1;|
| Placebo Comparator: placebo<br>2.5 mg/2mL placebo administered intranasally once daily | Drug: Placebo<br>* 2.5 mL of placebo delivered via Sinustar nebulizer device<br>* Other names: placebo comparator;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Computed Tomography Evidence of Less Sinus Disease | compare sinus CT pre-op (baseline) to one year after initiation of study drug Difference in pre and post scores by Lund-McKay scoring system are reported (1 year minus baseline) The Lund-Mackay scoring system was used to evaluate the extent and severity of sinusitis. The scale ranges from 0 (best possible outcome with complete lucency of all sinuses) to 24 (worst possible outcome with complete opacification of all sinuses) | baseline and 1 year | 
| Improvement in Appearance of Nasal Passages/Sinuses | periodic endoscopic photos of sinuses by ear-nose-throat (ENT) surgeon. The scale for scoring severity of disease ranges from 0 (best possible outcome) to 2 (worst possible outcome). independent blinded scoring by 2 surgeons difference in scores pre and post are reported (1 year minus baseline) | baseline and 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Chronic Sinusitis Survey Score | pre-surgery and end of trial (12 months) Reduction in scores (baseline minus 1 year) are recorded The chronic sinusitis survey consists of 6 questions, ranges from 0-24, a lower score indicates the best possible outcome. | baseline and 1 year | 
| Pulmonary Function | prior to surgery and end of study spirometry as measured by forced expiratory volume in 1 second (FEV1) percent predicted. The change over the course of the study (1 year minus baseline) is reported. A higher value indicates a better outcome. | baseline and 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Cystic fibrosis, chronic sinusitis, mucolytic