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

Genentech

Status and phase: Terminated; Phase 4

Conditions: Cystic Fibrosis

Treatments: Drug: Dornase alfa; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT00680316

Z4240g

Details and patient eligibility

About

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.

Enrollment: 3 patients

Sex: All

Ages: 3 to 5 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

---

3 participants in 2 patient groups, including a placebo group

Dornase alfa

Experimental group

Treatment:

Drug: Dornase alfa

Placebo

Placebo Comparator group

Treatment:

Drug: Placebo