Nasally Delivered Pulmozyme for Sinusitis in Cystic Fibrosis

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

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.

Study Overview

• Status: Completed
• Conditions: Sinusitis; Cystic Fibrosis
• Intervention / Treatment: Drug: Pulmozyme (dornase alfa); Drug: Placebo

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Vermont
    • Burlington, Vermont, United States, 05401
      • Fletcher Allen Health Care

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: University of Vermont
• Collaborators: Genentech, Inc.
• Investigators: Principal Investigator: Thomas Lahiri, MD, University of Vermont; Study Director: Sandra Diehl, MS, University of Vermont Medical Center

Publications and helpful links

General 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.
• Lahiri T, Herrington H, Diehl S, Landrigan G. The effect of intranasal dornase alfa on chronic sinusitis in patients with cystic fibrosis: a pilot study. Pediatr Pulmonol S35:354, 2012.

Study record dates

Study Major Dates

• Study Start: December 1, 2006
• Primary Completion (Actual): June 1, 2012
• Study Completion (Actual): June 1, 2012

Study Registration Dates

• First Submitted: December 26, 2006
• First Submitted That Met QC Criteria: December 26, 2006
• First Posted (Estimate): December 27, 2006

Study Record Updates

• Last Update Posted (Estimate): November 14, 2014
• Last Update Submitted That Met QC Criteria: November 12, 2014
• Last Verified: November 1, 2014

More Information

Terms related to this study

• Keywords: Cystic fibrosis; chronic sinusitis; mucolytic
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Infections; Respiratory Tract Infections; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Otorhinolaryngologic Diseases; Paranasal Sinus Diseases; Nose Diseases; Pancreatic Diseases; Fibrosis; Sinusitis; Cystic Fibrosis
• Other Study ID Numbers: Z3297S