Conditions: Cystic Fibrosis
Keywords: Pulmozyme CF
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 3
Healthy volunteers: no

Brief summary:
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.

Detailed description:

Arms:
arm 1: None arm 2: None

Interventions: intervention 1: Dornase alfa intervention 2: Placebo
Intervention descriptions: intervention 1: 2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days intervention 2: 2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days

Protocol document: