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

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE4

Total Enrollment

3 participants

Study Classification

INTERVENTIONAL

Study Start Date

2008-06-30

Study Completion Date

2009-05-31

Brief Summary

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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.

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Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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Dornase alfa

Group Type EXPERIMENTAL

Dornase alfa

Intervention Type DRUG

2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days

Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days

Interventions

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Dornase alfa

2.5 mL (2.5 mg) dornase alfa nebulized once daily for 16 (+/-2) days

Intervention Type DRUG

Placebo

2.5 mL (2.5 mg) placebo nebulized once daily for 16 (+/-2) days

Intervention Type DRUG

Other Intervention Names

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Pulmozyme

Eligibility Criteria

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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

Minimum Eligible Age

3 Years

Maximum Eligible Age

5 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No