Pulmozyme in Cystic Fibrosis With Sinusitis

July 17, 2017 updated by: Timothy Craig, Milton S. Hershey Medical Center

Z4770s, Use of Recombinant Human DNASE in Cystic Fibrosis Patients With Chronic Sinusitis to Prevent Acute Sinusitis Exacerbations and Improve Symptoms and Outcomes - A Pilot Study

The hypothesis is that the intranasal use of Pulmozyme will decrease the severity of sinusitis in Cystic Fibrosis and lead to an improved quality of life.

Study Overview

Status

Withdrawn

Detailed Description

The study will be a prospective enrollment of patients with CF and rhinosinusitis in a double-blind crossover study with nasal nebulized DNASE versus saline. Nebulization will be accomplished with PARI SinuStar nasal aerosol system nebulizer set powered by an air compressor. This will be a pilot study with each subject serving as their own control. Each arm of the study will be 3 months and randomization to active drug versus placebo will be generated without the knowledge of subject or researchers. A health care provider not involved in the care of the subject will be responsible for medication distribution and the randomization scheme. A daily diary will collect data for analysis. Visits will be arranged every 4 weeks at which time drug will be dispensed, adherence to daily diary and compliance to medication will be assessed. At each visit the SF-12, SNOT-20, Epworth Sleepiness Scale, Rhinitis Severity Scale, Nocturnal Rhinoconjunctivitis Quality of Life Specific Questionnaire (NRQLQ), Cystic Fibrosis Questionnaire-Revised, and Rhinosinusitis Quality of Life Survey will be utilized for acquisition of data (17-23). In addition, a Physician Global Assessment and a self administered patient rhinosinusitis VAS score (0-100 with 100 full recovery and 0 no effect) will be completed at each visit. To obtain objective data patients will have nasal endoscopic assessment at 4 different time points and nasal polyps and sinusitis rated on a scale as defined by Lund and Kennedy in the Ann Otol Laryngol 1995;104 (suppl 167):17-21. (24) Serial sinus CT scans will be avoided to decrease irradiation risk.

Subjects: A total of 12 patients will be enrolled in this pilot study, and recruitment will be through the Cystic Fibrosis Center at Penn State University, after IRB approval. All subjects must be over the age of 15 years (the age the investigators consider to be necessary to understand the consent and questionnaires required for the study), be of either gender and of any ethnicity. Subjects will be able to continue all presently used nasal and respiratory medications as long as the present therapeutic regimen has been used for one month prior to enrollment and these therapies have been used at a stable dose, method of distribution and without adverse events

Study Type

Interventional

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Unicersity

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  1. Patients with CF, age >15yo
  2. Chronic sinusitis with symptoms
  3. VAS score of at least 60 (0 to 100) reflecting symptoms associated with rhinosinusitis
  4. CT scan demonstrating non-complicated sinusitis (defined as rhinosinusitis without orbit perforation, osteomyelitis, peri-sinus cellulitis, or meningitis)

Exclusion Criteria:

  1. No evidence of sinusitis
  2. VAS score for rhinosinusitis less than 60 on a scale of 0 to 100
  3. Complicated sinusitis (defined as orbit perforation, osteomyelitis, peri-sinus cellulitis, meningitis complicating sinusitis)
  4. Prior adverse events or allergy to DNASE
  5. Unwilling to sign an approved IRB consent
  6. Immediate indication for sinus surgery
  7. Inability to adhere to therapy and understand and to complete questionnaires.
  8. Being pregnant will exclude a subject from participating and the subjects will be requested to take birth control methods if actively engaging in sex. Further more, subjects will be requested to inform the investigators if they become pregnant. Pregnancy test will be performed at screening.
  9. Active nursing of an infant will be an exclusion.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Crossover Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: PULMOZYME
active drug
• Each Pulmozyme single use ampule delivers 2.5 mL (2.5 mg) of the sterile solution to the nebulizer bowl. The aqueous solution contains 1.0 mg/mL dornase alfa, 0.15 mg/mL calcium chloride dihydrate, and 8.77 mg/mL sodium chloride.
Other Names:
  • Dornase alfa recombinant human deoxyribonuclease I (rhDNase)
Placebo Comparator: placebo
cross over to placebo
identical placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
1. To assess changes in quality of life of patients with Cystic Fibrosis while on DNASE as compared to placebo.
Time Frame: weeks
Sinusitis will be a significant burden for patients with CF. Use of nasal nebulized recombinant human DNASE will demonstrate positive changes in symptoms of rhinosinusitis and will improve the quality of life of patients with Cystic Fibrosis. In addition, subjects on active therapy will have a decrease in acute infections and a decrease in disease burden as compared to when the subject is on placebo therapy.
weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Collaborators

Investigators

  • Principal Investigator: Timothy Craig, DO, Penn State University

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

July 1, 2013

Primary Completion (Anticipated)

June 1, 2014

Study Completion (Anticipated)

June 1, 2015

Study Registration Dates

First Submitted

June 25, 2010

First Submitted That Met QC Criteria

July 1, 2010

First Posted (Estimate)

July 2, 2010

Study Record Updates

Last Update Posted (Actual)

July 21, 2017

Last Update Submitted That Met QC Criteria

July 17, 2017

Last Verified

November 1, 2012

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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