NCT01181622

Brief Summary

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

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Aug 2010

Shorter than P25 for phase_2

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2010

Completed
11 days until next milestone

First Submitted

Initial submission to the registry

August 12, 2010

Completed
1 day until next milestone

First Posted

Study publicly available on registry

August 13, 2010

Completed
2 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2010

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2010

Completed
Last Updated

January 13, 2015

Status Verified

December 1, 2014

Enrollment Period

2 months

First QC Date

August 12, 2010

Last Update Submit

December 29, 2014

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

  • Intolerability to study drug as measured by: oxyhemoglobin saturation, treatment-emergent cough, and new wheezes or crackles

    Day 1, Day 7

Secondary Outcomes (6)

  • 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

  • +1 more secondary outcomes

Study Arms (2)

denufosol tetrasodium Inhalation Solution

EXPERIMENTAL
Drug: denufosol tetrasodium Inhalation Solution

Placebo

PLACEBO COMPARATOR
Drug: 0.9% w/v sodium chloride solution

Interventions

denufosol tetrasodium Inhalation SolutionDRUG

60 mg by oral inhalation three times daily

denufosol tetrasodium Inhalation Solution
0.9% w/v sodium chloride solutionDRUG

0.9% w/v sodium chloride solution by oral inhalation three times daily

Placebo

Eligibility Criteria

Age2 Years - 4 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Officials

  • Lynn Smiley, MD

    Medical monitor

    STUDY DIRECTOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 12, 2010

First Posted

August 13, 2010

Study Start

August 1, 2010

Primary Completion

October 1, 2010

Study Completion

October 1, 2010

Last Updated

January 13, 2015

Record last verified: 2014-12