Nasally Delivered Pulmozyme for Sinusitis in Cystic Fibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00416182
Recruitment Status : Completed
First Posted : December 27, 2006
Results First Posted : November 14, 2014
Last Update Posted : November 14, 2014
Genentech, Inc.
Information provided by (Responsible Party):
Thomas Lahiri, University of Vermont

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Sinusitis Cystic Fibrosis Drug: Pulmozyme (dornase alfa) Drug: Placebo Phase 2

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  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: The Use of Nasally Delivered Pulmozyme in the Treatment of Sinusitis in Cystic Fibrosis Patients: A Pilot Study
Study Start Date : December 2006
Actual Primary Completion Date : June 2012
Actual Study Completion Date : June 2012

Resource links provided by the National Library of Medicine

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 Name: 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 Name: placebo comparator

Primary Outcome Measures :
  1. Computed Tomography Evidence of Less Sinus Disease [ Time Frame: baseline and 1 year ]
    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)

  2. Improvement in Appearance of Nasal Passages/Sinuses [ Time Frame: baseline and 1 year ]

    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)

Secondary Outcome Measures :
  1. Chronic Sinusitis Survey Score [ Time Frame: baseline and 1 year ]
    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.

  2. Pulmonary Function [ Time Frame: baseline and 1 year ]
    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.

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Ages Eligible for Study:   5 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00416182

United States, Vermont
Fletcher Allen Health Care
Burlington, Vermont, United States, 05401
Sponsors and Collaborators
University of Vermont
Genentech, Inc.
Principal Investigator: Thomas Lahiri, MD University of Vermont
Study Director: Sandra Diehl, MS University of Vermont Medical Center

Publications of Results:
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.

Responsible Party: Thomas Lahiri, Professor of Pediatrics, University of Vermont Identifier: NCT00416182     History of Changes
Other Study ID Numbers: Z3297S
First Posted: December 27, 2006    Key Record Dates
Results First Posted: November 14, 2014
Last Update Posted: November 14, 2014
Last Verified: November 2014

Keywords provided by Thomas Lahiri, University of Vermont:
Cystic fibrosis
chronic sinusitis

Additional relevant MeSH terms:
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Paranasal Sinus Diseases
Nose Diseases
Respiratory Tract Infections
Otorhinolaryngologic Diseases