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Nasally Delivered Pulmozyme for Sinusitis in Cystic Fibrosis

This study has been completed.
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Thomas Lahiri, University of Vermont
ClinicalTrials.gov Identifier:
NCT00416182
First received: December 26, 2006
Last updated: November 12, 2014
Last verified: November 2014
  Purpose

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 Intervention Phase
Sinusitis
Cystic Fibrosis
Drug: Pulmozyme (dornase alfa)
Drug: Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: The Use of Nasally Delivered Pulmozyme in the Treatment of Sinusitis in Cystic Fibrosis Patients: A Pilot Study

Resource links provided by NLM:


Further study details as provided by University of Vermont:

Primary Outcome Measures:
  • Computed Tomography Evidence of Less Sinus Disease [ Time Frame: baseline and 1 year ] [ Designated as safety issue: No ]
    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)

  • Improvement in Appearance of Nasal Passages/Sinuses [ Time Frame: baseline and 1 year ] [ Designated as safety issue: No ]

    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:
  • Chronic Sinusitis Survey Score [ Time Frame: baseline and 1 year ] [ Designated as safety issue: No ]
    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.

  • Pulmonary Function [ Time Frame: baseline and 1 year ] [ Designated as safety issue: No ]
    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.


Enrollment: 16
Study Start Date: December 2006
Study Completion Date: June 2012
Primary Completion Date: June 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.

  Eligibility

Ages Eligible for Study:   5 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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)
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00416182

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

No publications provided

Responsible Party: Thomas Lahiri, Professor of Pediatrics, University of Vermont
ClinicalTrials.gov Identifier: NCT00416182     History of Changes
Other Study ID Numbers: Z3297S
Study First Received: December 26, 2006
Results First Received: October 2, 2012
Last Updated: November 12, 2014
Health Authority: United States: Food and Drug Administration

Keywords provided by University of Vermont:
Cystic fibrosis
chronic sinusitis
mucolytic

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

ClinicalTrials.gov processed this record on November 25, 2014