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

Condition(s) targeted: Cystic Fibrosis

Intervention: Dornase alfa (Drug); Placebo (Drug)

Phase: Phase 4

Status: Terminated

Sponsored by: Genentech, Inc.

Official(s) and/or principal investigator(s):
Michelle Freemer, M.D., Study Director, Affiliation: Genentech, Inc.

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.

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

Study design: Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Primary outcome: Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment).

Secondary outcome:

Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10)

Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10)

Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers

Minimum age: 3 Years. Maximum age: 5 Years. Gender(s): Both.

Criteria:

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

Starting date: June 2008
Last updated: May 18, 2011