Study of Denufosol Inhalation Solution in Patients With Mild Cystic Fibrosis Lung Disease

A Multi-Center, Double-Blind, Placebo-Controlled Randomized, Efficacy and Safety Study of Denufosol Tetrasodium (INS37217) Inhalation Solution in Patients With Mild Cystic Fibrosis Lung Disease

The purpose of this trial is to evaluate the safety and effectiveness of a one dose strength of denufosol compared to placebo in patients with mild CF lung disease.

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis
• Intervention / Treatment: Drug: Placebo - 0.9% w/v sodium chloride solution; Drug: denufosol tetrasodium (INS37217) Inhalation Solution

• Study Type: Interventional
• Enrollment (Actual): 352
• Phase: Phase 3

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Have confirmed diagnosis of cystic fibrosis
• Have FEV1 of greater than or equal to 75% of predicted normal for age, gender, and height
• Be able to reproducibly perform spirometry maneuvers
• Be clinically stable for at least 4 weeks prior to screening

Exclusion Criteria:

• Have abnormal renal or liver function
• Have chest x-ray at screening suggesting clinically significant active pulmonary disease
• Be colonized with Burkholderia cepacia
• Have had a lung transplant

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
Experimental: 2	Drug: denufosol tetrasodium (INS37217) Inhalation Solution; Denufosol 60 mg is administered as an inhalation solution, three times daily for six months during the double-blind portion of the study. Subsequently, denufosol 60 mg is administered for an additional six months during the open label safety extension.
Placebo Comparator: 1; Placebo	Drug: Placebo - 0.9% w/v sodium chloride solution; 4.2 mL of solution, allowing delivery of approximately 4 mL into the nebulizer cup for nebulization, three times daily for six months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change in lung function	48 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Pulmonary exacerbation	48 weeks
Requirements for concomitant CF medications	48 weeks
Quality of Life	48 weeks

Collaborators and Investigators

• Sponsor: Merck Sharp & Dohme LLC

Publications and helpful links

General Publications

• Accurso FJ, Moss RB, Wilmott RW, Anbar RD, Schaberg AE, Durham TA, Ramsey BW; TIGER-1 Investigator Study Group. Denufosol tetrasodium in patients with cystic fibrosis and normal to mildly impaired lung function. Am J Respir Crit Care Med. 2011 Mar 1;183(5):627-34. doi: 10.1164/rccm.201008-1267OC. Epub 2010 Dec 17.

Study record dates

Study Major Dates

• Study Start: July 1, 2006
• Primary Completion (Actual): September 1, 2008
• Study Completion (Actual): September 1, 2008

Study Registration Dates

• First Submitted: July 25, 2006
• First Submitted That Met QC Criteria: July 26, 2006
• First Posted (Estimate): July 27, 2006

Study Record Updates

• Last Update Posted (Estimate): November 4, 2015
• Last Update Submitted That Met QC Criteria: October 9, 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; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Fibrosis; Lung Diseases; Pulmonary Fibrosis; Cystic Fibrosis; Pharmaceutical Solutions
• Other Study ID Numbers: 08-108

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No