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Pulmozyme in Cystic Fibrosis With Sinusitis

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified November 2012 by Penn State University
Sponsor:
Collaborator:
Genentech, Inc.
Information provided by (Responsible Party):
Timothy Craig, Penn State University
ClinicalTrials.gov Identifier:
NCT01155752
First received: June 25, 2010
Last updated: November 29, 2012
Last verified: November 2012
History of Changes
  Purpose

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.


Condition Intervention Phase
Sinusitis
Cystic Fibrosis
 Drug: Pulmozyme single use ampule
Drug: placebo
 Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Crossover Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Penn State University:

Primary Outcome Measures:
  • 1. To assess changes in quality of life of patients with Cystic Fibrosis while on DNASE as compared to placebo. [ Time Frame: weeks ] [ Designated as safety issue: No ]
    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.
Estimated Enrollment: 12
Study Start Date: July 2013
Estimated Study Completion Date: June 2015
Estimated Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: PULMOZYME
active drug
 Drug: Pulmozyme single use ampule
• 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 Name: Dornase alfa recombinant human deoxyribonuclease I (rhDNase)
Placebo Comparator: placebo
cross over to placebo
 Drug: placebo
identical placebo
Other Name: placebo

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

  Eligibility
Ages Eligible for Study:   12 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  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: NCT01155752

Contacts
Contact: Cathy Mende, NP 717-531-6525 cmende@hmc.psu.edu

Locations
United States, Pennsylvania
Penn State Unicersity Not yet recruiting
Hershey, Pennsylvania, United States, 17033
Contact: Timothy Craig, DO    717-531-6525    tcraig@psu.edu   
Principal Investigator: Timothy Craig, DO         
Sponsors and Collaborators
Penn State University
Genentech, Inc.
Investigators
Principal Investigator: Timothy Craig, DO Penn State University
  More Information

No publications provided

Responsible Party: Timothy Craig, Principal Investigator, Penn State University
ClinicalTrials.gov Identifier: NCT01155752     History of Changes
Other Study ID Numbers: IRB#33344
Study First Received: June 25, 2010
Last Updated: November 29, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by Penn State University:
sinusitis
cystic fibrosis
therapy

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Sinusitis
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 March 01, 2015