A Phase IV Multicenter, Randomized, Open Label, Two-Period, Crossover Study in Patients With Cystic Fibrosis to Evaluate the Comparable Efficacy and Safety of Pulmozyme Delivered by the eRapid Nebulizer System
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 99
- 主要终点
- Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)
研究概览
简要总结
This phase IV, multicenter, randomized, open-label, two-period crossover study will evaluate the comparable efficacy and safety of Pulmozyme (dornase alfa) delivered by the eRapid nebulizer system in patients with cystic fibrosis. Patients who have been receiving Pulmozyme once daily chronically for at least 6 months will continue to receive Pulmozyme once daily for a run-in period of 2 weeks using the Pari LC Plus nebulizer. Patients will then be randomized to receive in a crossover design Pulmozyme once daily for two treatment periods of 2 weeks each using either the Pari LC Plus or the eRapid nebulizer. Anticipated time on study treatment is 6 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female patients, >/= 6 years of age
- •Confirmed diagnosis of cystic fibrosis (CF)
- •Receiving Pulmozyme once daily chronically for treatment of CF for at least 6 months prior to screening
- •Percent predicted FEV1 >/= 40% at screening based on the Wang (males < 18 years, females < 16 years) or Hankinson (males >/= 18 years, females >/= 16 years) standardized equations
- •Able to reproducibly perform spirometry testing and comply with study assessments
排除标准
- •An acute respiratory infection or pulmonary exacerbation within 4 weeks prior to randomization
- •Initiation of any new chronic therapy (e.g. inhaled corticosteroids, inhaled oral antibiotics, high-dose ibuprofen, hypertonic saline, ivacaftor) for respiratory disease within 4 weeks prior to randomization
- •Changes in chest physiotherapy schedule within 4 weeks prior to randomization
- •Hospitalization within 4 weeks prior to randomization
- •Planned hospitalization during the 6-week study
- •History of organ transplantation
- •Participation in an investigational drug or device study within 30 day prior to screening
研究组 & 干预措施
eRapid Nebulizer
Dornase alfa (Pulmozyme®) inhaled once daily by the Pari eRapid nebulizer for 2 weeks.
干预措施: dornase alfa [Pulmozyme®] (Drug)
Jet Nebulizer
Dornase alfa (Pulmozyme®) inhaled once daily by the Pari LC Plus jet nebulizer for 2 weeks.
干预措施: dornase alfa [Pulmozyme®] (Drug)
结局指标
主要结局
Stability of Lung Function: Percent Predicted Forced Expiratory Volume in 1 Second (FEV1)
时间窗: At the end of each 2-week treatment period
Spirometry was performed according to American Thoracic Society standards. FEV1 is the amount of air that is forced out of the lungs in one second and was measured at the end of each 2-week treatment period. The percent predicted FEV1 was calculated as: Percent predicted FEV1 =FEV1 (L) / Predicted FEV1 (L) ×100.
Safety: Number of Participants With Adverse Events During Each Treatment Period
时间窗: 4 Weeks
An adverse event was considered any unfavorable and unintended sign, symptom, or disease associated with the use of the study drug, whether or not considered related to the study drug. Preexisting conditions that worsened during the study and laboratory or clinical tests that resulted in a change in treatment or discontinuation from study drug were reported as adverse events.
次要结局
未报告次要终点