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) (DEFY)

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.

Study Overview

• Status: Terminated
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: Denufosol tetrasodium Inhalation Solution

• Study Type: Interventional
• Enrollment (Actual): 308
• Phase: Phase 3

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Denufosol tetrasodium Inhalation Solution	Drug: Denufosol tetrasodium Inhalation Solution; 60 mg by oral inhalation three times daily

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	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

Collaborators and Investigators

• Sponsor: Merck Sharp & Dohme LLC
• Investigators: Study Director: Lynn Smiley, MD, Medical Monitor

Study record dates

Study Major Dates

• Study Start: February 1, 2009
• Primary Completion (ACTUAL): February 1, 2011
• Study Completion (ACTUAL): February 1, 2011

Study Registration Dates

• First Submitted: February 18, 2009
• First Submitted That Met QC Criteria: February 18, 2009
• First Posted (ESTIMATE): February 19, 2009

Study Record Updates

• Last Update Posted (ESTIMATE): November 6, 2015
• Last Update Submitted That Met QC Criteria: October 15, 2015
• Last Verified: October 1, 2015

More Information

Terms related to this study

• 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: P08642; 08-114