Cystic Fibrosis
Conditions
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
Interventions
60 mg by oral inhalation three times daily
DRUG0.9% w/v sodium chloride solution
0.9% w/v sodium chloride solution by oral inhalation three times daily
Sponsors
Merck Sharp & Dohme LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
2 Years to 4 Years
Healthy volunteers
No
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
Design outcomes
Primary
| 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
| Measure | Time frame |
|---|---|
| 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 oxyhemoglobin saturation 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 |
| Mean change from pre-dose in pulse and respiratory rate at defined times post-dose | Day 1, Day 7 |
Outcome results
None listed