A Safety and Tolerability Study of Denufosol in 2-4 Year Olds (REACH-1)

A Phase 2, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, 7-Day Safety and Tolerability Study of Denufosol Tetrasodium Inhalation Solution Administered Via PARI LC® Star in Patients 2 to 4 Years of Age With Cystic Fibrosis

The purpose of this study is to evaluate the safety and tolerability of denufosol 60 mg TID in pediatric CF patients 2 to 4 years of age

Study Overview

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

• Study Type: Interventional
• Enrollment (Actual): 25
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 4 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Have a confirmed diagnosis of CF
• Have oxyhemoglobin saturation ≥ 95% prior to randomization

Exclusion Criteria:

• Have acute intercurrent respiratory infection (cough, wheezing, or new

rhinorrhea or nasal congestion)

• Have any significant medical condition not related to CF
• Unable to discontinue use of hypertonic saline

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: DOUBLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
PLACEBO_COMPARATOR: Placebo	Drug: 0.9% w/v sodium chloride solution; 0.9% w/v sodium chloride solution by oral inhalation three times daily
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
Intolerability to study drug as measured by: oxyhemoglobin saturation, treatment-emergent cough, and new wheezes or crackles	Day 1, Day 7

Secondary Outcome Measures

Outcome Measure	Time Frame
Mean change from pre-dose in oxyhemoglobin saturation at defined times post-dose	Day 1, Day 7
Mean change from baseline in oxyhemoglobin saturation	Day 7
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and/or withdrawals due to TEAEs	Day 7
Mean change from pre-dose in pulse and respiratory rate at defined times post-dose	Day 1, Day 7
Mean change from baseline in pulse and respiratory rate	Day 7
Incidence of new medical condition(s) or worsening of previous medical condition(s) from baseline	Day 7

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start: August 1, 2010
• Primary Completion (ACTUAL): October 1, 2010
• Study Completion (ACTUAL): October 1, 2010

Study Registration Dates

• First Submitted: August 12, 2010
• First Submitted That Met QC Criteria: August 12, 2010
• First Posted (ESTIMATE): August 13, 2010

Study Record Updates

• Last Update Posted (ESTIMATE): January 13, 2015
• Last Update Submitted That Met QC Criteria: December 29, 2014
• Last Verified: December 1, 2014

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; Pharmaceutical Solutions
• Other Study ID Numbers: P08643; 08-116