A Phase IV, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial of Pulmozyme® in 3- to 5-Year-Old Patients With Cystic Fibrosis
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 入组人数
- 3
- 主要终点
- Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment).
研究概览
简要总结
This was a Phase IV, multicenter, randomized, double-blind, placebo-controlled trial designed to evaluate the effect of Pulmozyme on pulmonary function, health-related quality of life (HRQOL), and respiratory symptoms in 3- to 5-year-old children with cystic fibrosis (CF). Approximately 40 patients were planned to be enrolled in this study. However, only 3 patients were eligible for random allocation and received treatment: 1 patient in the Pulmozyme group and 2 patients in the placebo group. All 3 patients completed the study assessments but did not have usable pulmonary function test (PFT) data.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 3 Years 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed Informed Consent Form
- •Aged 3-5 years
- •Diagnosis of cystic fibrosis
排除标准
- •Children taking scheduled inhaled Pulmozyme or hypertonic saline within 56 days prior to Visit 1 or any Pulmozyme in the 28 days before Visit 1
- •Involvement in a clinical intervention trial within the 4 weeks prior to Visit 1
- •Use of an investigational drug or device within 28 days prior to Visit 1
- •Any other condition that might increase the risk of participation to the patient in the judgement of the investigator
研究组 & 干预措施
Dornase alfa
干预措施: Dornase alfa (Drug)
Placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Reactance at 8 Hz (Xrs8) From Visit 2 to Visit 3 (Change From Baseline at Visit 2 to Visit 3, After Study Drug Treatment).
时间窗: from Visit 2 to Visit 3 (16 +/- 2 days)
The fundamental principle of forced oscillometry is that lung function can be assessed by measuring changes in pressure and flow in response to external pressure applied at the airway opening. Reactance is complex measure that incorporates the changes in pressure and volume and the rate of these changes in response to pressure oscillations at a specific frequency. (8Hz was used for the primary endpoint). Reactance is thought to reflect the elastic properties of the lung.
次要结局
- Change in Resistance at 4, 6, 8, and 10 Hz (Rrs4, Rrs6, Rrs8, and Rrs10)(from Visit 2 to Visit 3 (16 +/- 2 days))
- Change in Reactance at 4, 6, and 10 Hz (Xrs4, Xrs6, and Xrs10)(from Visit 2 to Visit 3 (16 +/- 2 days))
- Change in Respiratory Symptom Domain Score From the Cystic Fibrosis Questionnaire Revised (CFQ-R) for Parents of Preschoolers and for Preschoolers(from Visit 2 to Visit 3 (16 +/- 2 days))