A Study of Pulmozyme® (Dornase Alpha) in 3- to 5-Year-Old Patients With Cystic Fibrosis

A Phase IV, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme® in 3- to 5-Year-Old Patients With Cystic Fibrosis

This was a Phase IV, multicenter, randomized, double-blind, placebo-controlled trial designed to evaluate the effect of Pulmozyme on pulmonary function, health-related quality of life (HRQOL), and respiratory symptoms in 3- to 5-year-old children with cystic fibrosis (CF). Approximately 40 patients were planned to be enrolled in this study. However, only 3 patients were eligible for random allocation and received treatment: 1 patient in the Pulmozyme group and 2 patients in the placebo group. All 3 patients completed the study assessments but did not have usable pulmonary function test (PFT) data.

Inclusion Criteria:

• Signed Informed Consent Form
• Aged 3-5 years
• Diagnosis of cystic fibrosis

Exclusion Criteria:

• Children taking scheduled inhaled Pulmozyme or hypertonic saline within 56 days prior to Visit 1 or any Pulmozyme in the 28 days before Visit 1
• Involvement in a clinical intervention trial within the 4 weeks prior to Visit 1
• Use of an investigational drug or device within 28 days prior to Visit 1
• Any other condition that might increase the risk of participation to the patient in the judgement of the investigator