Pulmozyme in Cystic Fibrosis With Sinusitis

• ClinicalTrials.gov Identifier: NCT01155752
• Recruitment Status: Withdrawn
• First Posted: July 2, 2010
• Last Update Posted: July 21, 2017
• Sponsor: Milton S. Hershey Medical Center
• Collaborator: Genentech, Inc.
• Information provided by (Responsible Party): Timothy Craig, Milton S. Hershey Medical Center

Tracking Information

• First Submitted Date: June 25, 2010
• First Posted Date: July 2, 2010
• Last Update Posted Date: July 21, 2017
• Study Start Date: July 2013
• Estimated Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (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: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Pulmozyme in Cystic Fibrosis With Sinusitis
• 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
• 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: Interventional
• Study Phase: Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Crossover Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment

Condition

• Sinusitis
• Cystic Fibrosis

Intervention

• 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

• Experimental: PULMOZYME
  active drug
  Intervention: Drug: Pulmozyme single use ampule
• Placebo Comparator: placebo
  cross over to placebo
  Intervention: Drug: placebo

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Withdrawn
• Actual Enrollment (submitted: July 17, 2017): 0
• Original Estimated Enrollment (submitted: July 1, 2010): 12
• Estimated Study Completion Date: June 2015
• Estimated Primary Completion Date: June 2014 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years to 65 Years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01155752
• Other Study ID Numbers: IRB#33344
• Has Data Monitoring Committee: No
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: Timothy Craig, Milton S. Hershey Medical Center
• Study Sponsor: Milton S. Hershey Medical Center
• Collaborators: Genentech, Inc.

Investigators

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

• PRS Account: Milton S. Hershey Medical Center
• Verification Date: November 2012