Working… Menu

Pulmozyme in Cystic Fibrosis With Sinusitis

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: NCT01155752
Recruitment Status : Withdrawn (Never funded)
First Posted : July 2, 2010
Last Update Posted : July 21, 2017
Genentech, Inc.
Information provided by (Responsible Party):
Timothy Craig, Milton S. Hershey Medical Center

Tracking Information
First Submitted Date  ICMJE June 25, 2010
First Posted Date  ICMJE July 2, 2010
Last Update Posted Date July 21, 2017
Study Start Date  ICMJE July 2013
Estimated Primary Completion Date June 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: July 1, 2010)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Pulmozyme in Cystic Fibrosis With Sinusitis
Official Title  ICMJE 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
Brief Summary 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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE
  • Sinusitis
  • Cystic Fibrosis
Intervention  ICMJE
  • 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)
  • Drug: placebo
    identical placebo
Study Arms  ICMJE
  • Experimental: PULMOZYME
    active drug
    Intervention: Drug: Pulmozyme single use ampule
  • Placebo Comparator: placebo
    cross over to placebo
    Intervention: Drug: placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Withdrawn
Actual Enrollment  ICMJE
 (submitted: July 17, 2017)
Original Estimated Enrollment  ICMJE
 (submitted: July 1, 2010)
Estimated Study Completion Date  ICMJE June 2015
Estimated Primary Completion Date June 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years to 65 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT01155752
Other Study ID Numbers  ICMJE IRB#33344
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Timothy Craig, Milton S. Hershey Medical Center
Study Sponsor  ICMJE Milton S. Hershey Medical Center
Collaborators  ICMJE Genentech, Inc.
Investigators  ICMJE
Principal Investigator: Timothy Craig, DO Penn State University
PRS Account Milton S. Hershey Medical Center
Verification Date November 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP