Pulmozyme in Cystic Fibrosis With Sinusitis

NCT01155752 | Withdrawn Phase 3

• 1 other identifier: IRB#33344
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Sinusitis; Cystic Fibrosis

Keywords

sinusitis; cystic fibrosis; therapy

Outcome Measures

Primary Outcomes (1)

• 1. To assess changes in quality of life of patients with Cystic Fibrosis while on DNASE as compared to placebo.

  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

Study Arms (2)

PULMOZYME

EXPERIMENTAL

active drug

Drug: Pulmozyme single use ampule

placebo

PLACEBO COMPARATOR

cross over to placebo

Drug: placebo

Interventions

Pulmozyme single use ampule 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.

Also known as: Dornase alfa recombinant human deoxyribonuclease I (rhDNase)

PULMOZYME

placebo DRUG

identical placebo

placebo

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• No evidence of sinusitis
• VAS score for rhinosinusitis less than 60 on a scale of 0 to 100
• Complicated sinusitis (defined as orbit perforation, osteomyelitis, peri-sinus cellulitis, meningitis complicating sinusitis)
• Prior adverse events or allergy to DNASE
• Unwilling to sign an approved IRB consent
• Immediate indication for sinus surgery
• Inability to adhere to therapy and understand and to complete questionnaires.
• 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.

Sponsors & Collaborators

• Milton S. Hershey Medical Center: lead
• Genentech, Inc.: collaborator

Study Sites (1)

Penn State Unicersity

Hershey, Pennsylvania, 17033, United States

Location

MeSH Terms

Conditions

Sinusitis; Cystic Fibrosis

Interventions

dornase alfa

Condition Hierarchy (Ancestors)

Respiratory Tract Infections; Infections; Paranasal Sinus Diseases; Nose Diseases; Respiratory Tract Diseases; Otorhinolaryngologic Diseases; Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Timothy Craig, DO

  Penn State University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 25, 2010
• First Posted: July 2, 2010
• Study Start: July 1, 2013
• Primary Completion: June 1, 2014
• Study Completion: June 1, 2015
• Last Updated: July 21, 2017

Record last verified: 2012-11

Locations

US (1)