Study 08-114 Open-label Extension of Study 08-110 - A Multi-Center Study of Denufosol Tetrasodium Inhalation Solution in Patients With Cystic Fibrosis Lung Disease (P08642)

Study Overview
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Brief Summary
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The purpose of this study is to obtain data on the potential of long-term treatment with denufosol to improve the clinical course of CF lung disease in patients with mildly impaired lung function and to provide CF patients who completed study 08-110 continued access to denufosol.

Conditions
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Cystic Fibrosis

Intervention / Treatment
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* Drug: Denufosol tetrasodium Inhalation Solution


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Completed Study 08-110 Exclusion Criteria: A female patient will not be eligible for the study if she is of childbearing potential and is pregnant, lactating, and/or not practicing an acceptable method of birth control (e.g., abstinence, hormonal or barrier methods, partner sterilization, or intrauterine device). Have a condition that might affect compliance with study procedures

Ages Eligible for Study
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Minimum Age: 5 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Denufosol tetrasodium Inhalation Solution<br> | Drug: Denufosol tetrasodium Inhalation Solution<br>* 60 mg by oral inhalation three times daily<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Rate of change in lung function, as measured by change in FEV1 |  | 160 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in absolute FEV1 from baseline |  | Weeks 12, 24, 36, 48, 72, 96, 120, 144, and 160 | 
| Change in FEV1 percent predicted value from baseline |  | Weeks 12, 24, 36, 48, 72, 96, 120, 144, and 160 | 
| Change in FEF25%-75% and forced vital capacity (FVC) from baseline |  | Weeks 12, 24, 36, 48, 72, 96, 120, 144, and 160 | 
| Incidence of pulmonary exacerbations |  | 160 Weeks | 
| Changes in Pseudomonas aeruginosa status |  | 160 Weeks | 
| Number of days of intravenous (IV) antibiotic use for a respiratory complaint |  | 160 Weeks | 
| Incidence of hospitalizations/Emergency room (ER) visits for a respiratory complaint |  | 160 Weeks | 
| Number of days hospitalized for a respiratory complaint |  | 160 Weeks | 
| Incidence of treatment emergent adverse events (AEs), serious adverse events (SAEs) and withdrawals due to AEs |  | 160 Weeks | 
| Incidence of patients with significant decrease in absolute FEV1 at any visit from baseline |  | 160 Weeks | 
| Changes in physical exam findings including height, weight, and body mass index from baseline |  | Weeks 48, 96, and 160 | 
| Changes in hematology, serum chemistry, and urinalysis parameters from baseline |  | Weeks 24, 48, 72, 96, 120, 144, and 160 | 
| Incidence of hospitalizations/ER visits |  | 160 Weeks | 
| Incidence of early discontinuations from the study |  | 160 Weeks |