Effectiveness of Pulmozyme in Infants With Cystic Fibrosis

• Status: Completed
• Trial end date: 2016-07-01
• Target enrollment: 0
• Participant gender: All

Summary

This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Nationwide Children's Hospital
• Collaborator: Genentech, Inc.

Criteria

Inclusion Criteria:

• Age < 30 months

• Diagnosis of CF based on clinical features consistent with CF as well as 1 of the 2 following criteria: a) two sweat chlorides >60 mEq/L (by quantitative pilocarpine iontophoresis), b) genotype with 2 identifiable mutations consistent with CF.

• Informed consent by parent or legal guardian

Exclusion Criteria:

• Previous treatment with Pulmozyme

• Hospitalization or treatment with IV antibiotics with 14 days of initial study visit

• Acute intercurrent respiratory infection, defined as any of the following symptoms within the preceding 48 hours: 1) fever > 38 degrees C, 2) new onset of coryza or other upper respiratory symptoms, 3) increase in cough, wheezing, or respiratory rate

• History of adverse reaction to sedation

• Oxyhemoglobin saturation <90% on room air

• Severe upper airway obstruction as determined by site PI (severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed obstructive sleep apnea)

• Hemodynamically significant congenital heart disease or diagnosed arrhythmias

• History of hemoptysis

• History of previous pulmonary air leak (pneumothorax)

• Diagnosed seizure disorder necessitating current anticonvulsive therapy. A history of febrile seizures is not an exclusion criterion.

• Use of Investigational drug(s) within 60 days or 5 half-lives of enrollment in this study.

• Known allergy to Chinese Hamster Ovary-derived biological products or any component of the placebo or active drug formulations.