Phase 2
N=24
Effectiveness of Pulmozyme in Infants With Cystic Fibrosis
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT00179998 ↗Enrolled (actual)
24
Serious AEs
4.2%
Results posted
May 2019
Primary outcome: Primary: Chest CT (High Resolution Computed Tomography (HRCT) Score) — 3.7; 6.1 Score points
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Recombinant Human DNase (Pulmozyme) (Drug); Placebos (Drug)
- Age
- Pediatric · 0+ yrs
- Sex
- All
- Sponsor
- Nationwide Children's Hospital
- Primary completion
- Oct 2008
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Chest CT (High Resolution Computed Tomography (HRCT) Score) |
3.7; 6.1 | — |
| PRIMARY Infant Pulmonary Function Tests (FEV0.5) |
-0.1; -0.2 | — |
| SECONDARY Antibiotic Treatment Days |
31.7; 36.3 | — |
Summary
This is a study to find out whether Pulmozyme is effective for clearing mucus from the airways of children with cystic fibrosis less than 3 ½ years of age.
Eligibility Criteria
Inclusion Criteria
- Age 60 mEq/L (by quantitative pilocarpine iontophoresis), b) genotype with 2 identifiable mutations consistent with CF.
- Informed consent by parent or legal guardian
Exclusion Criteria
- Previous treatment with Pulmozyme
- Hospitalization or treatment with IV antibiotics with 14 days of initial study visit
- Acute intercurrent respiratory infection, defined as any of the following symptoms within the preceding 48 hours: 1) fever > 38 degrees C, 2) new onset of coryza or other upper respiratory symptoms, 3) increase in cough, wheezing, or respiratory rate
- History of adverse reaction to sedation
- Oxyhemoglobin saturation <90% on room air
- Severe upper airway obstruction as determined by site PI (severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed obstructive sleep apnea)
- Hemodynamically significant congenital heart disease or diagnosed arrhythmias
- History of hemoptysis
- History of previous pulmonary air leak (pneumothorax)
- Diagnosed seizure disorder necessitating current anticonvulsive therapy. A history of febrile seizures is not an exclusion criterion.
- Use of Investigational drug(s) within 60 days or 5 half-lives of enrollment in this study.
- Known allergy to Chinese Hamster Ovary-derived biological products or any component of the placebo or active drug formulations.
Data sourced from ClinicalTrials.gov (NCT00179998). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.