A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 77
- 试验地点
- 48
- 主要终点
- Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
研究概览
简要总结
The purpose of this study is to evaluate the efficacy, safety, pharmacodynamic (PD) and pharmacokinetic (PK) effect of VX-561.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D
- •On ivacaftor therapy
- •FEV1 value ≥40% and ≤100% of predicted mean for age, sex, and height
排除标准
- •History of clinically significant cirrhosis with or without portal hypertension
- •History of solid organ or hematological transplantation
- •Lung infection with organisms associated with a more rapid decline in pulmonary status
- •Other protocol defined Inclusion/Exclusion criteria may apply
研究组 & 干预措施
Ivacaftor
Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.
干预措施: IVA (Drug)
Ivacaftor
Participants received IVA 150 milligrams (mg) orally every 12 hours (q12h) in the treatment period for 12 weeks.
干预措施: Placebo (Drug)
VX-561: 25 mg
Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.
干预措施: VX-561 (Drug)
VX-561: 25 mg
Participants received VX-561 25 mg orally daily (qd) in the treatment period for 12 weeks.
干预措施: Placebo (Drug)
VX-561: 50 mg
Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
干预措施: VX-561 (Drug)
VX-561: 50 mg
Participants received VX-561 50 mg orally qd in the treatment period for 12 weeks.
干预措施: Placebo (Drug)
VX-561: 150 mg
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
干预措施: VX-561 (Drug)
VX-561: 150 mg
Participants received VX-561 150 mg orally qd in the treatment period for 12 weeks.
干预措施: Placebo (Drug)
VX-561: 250 mg
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
干预措施: VX-561 (Drug)
VX-561: 250 mg
Participants received VX-561 250 mg orally qd in the treatment period for 12 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
时间窗: From Baseline at Week 12
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
次要结局
- Absolute Change in Sweat Chloride (SwCl)(From Baseline at Week 12)
- Observed Pre-Dose Concentration (Ctrough) of VX-561 and Its Metabolites (M1-VX-561 and M6-VX-561) and IVA and Its Metabolites (M1-IVA and M6-IVA)(At Week 4)
- Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(Baseline up to Week 16)