A Phase 2, Randomized, Double-Blind, Placebo Controlled, Parallel-Group, Exploratory Study to Evaluate Effects of VX-661 in Combination With Ivacaftor on Lung and Extrapulmonary Systems in Subjects Aged 18 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 34
- 主要终点
- Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28
研究概览
简要总结
To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA) (TEZ/IVA) in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.
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
- •Male and female participants, homozygous for the F508del CFTR mutation
- •Confirmed diagnosis of CF by sweat chloride testing
- •Forced Expiratory Volume in 1 Second (FEV1) ≥40% and ≤90% of predicted normal for age, sex, and height at Screening Visit
- •Stable CF disease as judged by the investigator.
排除标准
- •History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.
- •An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1
- •History or evidence of clinically significant findings on ophthalmologic examination during the Screening Period.
- •History of solid organ or hematological transplantation
- •Pregnant or nursing females
- •Participants who have had radiation exposure within 1 year before the first mucociliary clearance (MCC) procedure that would cause them to exceed federal regulations by participating in this study
- •In the opinion of the investigator, unable to adequately perform inhalation maneuvers during the MCC procedures
研究组 & 干预措施
Placebo
Participants received placebo matched to tezacaftor (TEZ)/ivacaftor (IVA) fixed dose combination (FDC) tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days.
干预措施: Tezacaftor/Ivacaftor matching placebo (Drug)
Placebo
Participants received placebo matched to tezacaftor (TEZ)/ivacaftor (IVA) fixed dose combination (FDC) tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 29 days.
干预措施: Ivacaftor matching placebo (Drug)
TEZ/IVA
Participants received 100 milligram (mg) TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days.
干预措施: Tezacaftor/Ivacaftor (Drug)
TEZ/IVA
Participants received 100 milligram (mg) TEZ/150 mg IVA FDC tablet orally once daily in the morning followed by 150 mg IVA tablet orally once daily in the evening for 29 days.
干预措施: Ivacaftor (Drug)
结局指标
主要结局
Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28
时间窗: Baseline, Day 28
MCC was assessed using an imaging technique that enables the tracking of mucus within the airways. MCC was expressed as the percentage of whole-lung clearance through 60 minutes at Baseline and Day 28.
次要结局
- Absolute Change From Baseline in Small-bowel Area Under the Curve (AUC) Over 1-minute Mean pH Increments at Day 29(Baseline, Day 29)
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)(Baseline up to Day 57)
- Absolute Change From Baseline in Sweat Chloride at Day 29(Baseline, Day 29)
- Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28(Baseline, Day 28)