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• Recruitment Status: Withdrawn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

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Sinusitis; Cystic Fibrosis	Drug: Pulmozyme single use ampule; Drug: placebo	Phase 3

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 Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 0 participants
• Allocation: Randomized
• Intervention Model: Crossover Assignment
• Masking: Quadruple (Participant, Care Provider, 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
• Study Start Date: July 2013
• Estimated Primary Completion Date: June 2014
• Estimated Study Completion Date: June 2015

Arms and Interventions

Arm	Intervention/treatment
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

Outcome Measures

Primary Outcome Measures :

1. 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.

Eligibility Criteria

• Ages Eligible for Study: 12 Years to 65 Years (Child, Adult, Older Adult)
• Sexes Eligible for Study: All
• 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

Locations

United States, Pennsylvania

Penn State Unicersity

Hershey, Pennsylvania, United States, 17033

Sponsors and Collaborators

Milton S. Hershey Medical Center

Genentech, Inc.

Investigators

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

More Information

• Responsible Party: Timothy Craig, Principal Investigator, Milton S. Hershey Medical Center
• ClinicalTrials.gov Identifier: NCT01155752
• Other Study ID Numbers: IRB#33344
• First Posted: July 2, 2010
• Last Update Posted: July 21, 2017
• Last Verified: November 2012

Keywords provided by Timothy Craig, Milton S. Hershey Medical Center:

sinusitis; cystic fibrosis; therapy

Additional relevant MeSH terms:

Cystic Fibrosis; Sinusitis; Fibrosis; Pathologic Processes; Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Infant, Newborn, Diseases; Paranasal Sinus Diseases; Nose Diseases; Respiratory Tract Infections; Otorhinolaryngologic Diseases