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.

Study Overview

• Status: Terminated
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: Dornase alfa; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 3
• Phase: Phase 4

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 5 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; 2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days
Experimental: Dornase alfa	Drug: Dornase alfa; 2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days; Other Names: Pulmozyme

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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).	The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.	from Visit 2 to Visit 3 (16 +/- 2 days)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10)	The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.	from Visit 2 to Visit 3 (16 +/- 2 days)
Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10)	The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Resistance is complex measure that incorporates the lack of changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (10Hz was used for the secondary endpoint).	from Visit 2 to Visit 3 (16 +/- 2 days)
Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers	The CFQ-R for Preschoolers and the CFQ-R for Parents of Preschoolers was designed specifically to measure the impact of CF for patients with a diagnosis of CF. Each question is answered using a 4-point Likert scale. In order to calculate the domain/symptom scale scores, the following algorithm is followed; Re-number items which have been reverse coded; Calculate the mean of the items to be included. If more than half of the items are missing, then the score is considered missing; Re-scale to result in a scaled score which ranges from 0 to 100, with higher scores indicating better health	from Visit 2 to Visit 3 (16 +/- 2 days)

Collaborators and Investigators

• Sponsor: Genentech, Inc.
• Investigators: Study Director: Michelle Freemer, M.D., Genentech, Inc.

Study record dates

Study Major Dates

• Study Start: June 1, 2008
• Primary Completion (Actual): May 1, 2009
• Study Completion (Actual): May 1, 2009

Study Registration Dates

• First Submitted: May 16, 2008
• First Submitted That Met QC Criteria: May 19, 2008
• First Posted (Estimate): May 20, 2008

Study Record Updates

• Last Update Posted (Estimate): January 12, 2017
• Last Update Submitted That Met QC Criteria: November 25, 2016
• Last Verified: November 1, 2016

More Information

Terms related to this study

• Keywords: CF; Pulmozyme
• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Cystic Fibrosis
• Other Study ID Numbers: Z4240g