A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled, Parallel-group Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 Administered Orally Once-Daily for 48 Weeks in Adult Patients With Cystic Fibrosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 200
- 试验地点
- 75
- 主要终点
- Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1)
研究概览
简要总结
This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF.
详细描述
This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF. A total of 195 pulmonary CF patients that meet all the inclusion and no exclusion criteria and provide written informed consent will be randomized to receive 50 mg CTX-4430, 100 mg CTX-4430, or placebo in a 1:1:1 ratio. Follow-up visits will be conducted approximately every 4 weeks from Week 4 to Week 52 (4 weeks after completion of treatment).
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 30 Years（Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Forced expiratory volume at one second (FEV1) ≥50 percent predicted at Screening
- •At least 1 pulmonary exacerbation in the 12 months before Screening
排除标准
- •Pregnant or nursing women
- •Medical condition that is unstable, could be adversely impacted by participation in the study, or could impact assessment of the study results
- •History of organ transplantation
- •History of alcoholism or drug abuse within 2 years before Screening
- •Regular use of a high-dose NSAID within 60 days before Screening
研究组 & 干预措施
50 mg CTX-4430
Once daily oral capsule for 48 weeks
干预措施: CTX-4430 (Drug)
100 mg CTX-4430
Once daily oral capsule for 48 weeks
干预措施: CTX-4430 (Drug)
Matching Placebo
Once daily oral capsule for 48 weeks
干预措施: Placebo (Drug)
结局指标
主要结局
Difference From Placebo in Absolute Change From Baseline in Forced Expiratory Volume in 1 Second Percent Predicted (ppFEV1)
时间窗: Baseline, Week 48
Difference from Placebo in absolute change from Baseline at Week 48 was assessed for FEV1 percent predicted.
次要结局
- Change From Baseline at 48 Weeks for Forced Vital Capacity Percent Predicted (FVC) and FEF25-75% (Forced Expiratory Flow During the Middle Half of the Forced Vital Capacity) Percent Predicted(Baseline, Week 48)
- Change From Baseline for C-reactive Protein (Hs-CRP)(Baseline, Week 48)
- Number of Pulmonary Exacerbations Through 48 Weeks(Week 48)
- Subjects Without a Pulmonary Exacerbation While in the Study(Week 48)
- Change From Baseline for Specified Biomarkers(Baseline, Week 48)
- Hazard Ratio Pulmonary Exacerbation While in the Study(Week 48)
- Relative Change (Percent Change) From Baseline in ppFEV1(Baseline, Week 48)