A Phase 4, Open-label Treatment, Randomized, Multicenter, 2-arm, Parallel-group, Pilot Study of Adherence to Lumacaftor/Ivacaftor in CF Subjects Homozygous for the F508del CFTR Mutation
试验速览
- 阶段
- 4 期
- 状态
- 终止
- 入组人数
- 24
- 主要终点
- Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment
研究概览
简要总结
The study will evaluate the impact of smart adherence technology for monitoring on lumacaftor/ivacaftor (LUM/IVA) adherence rates among subjects 16 years of age and older with Cystic Fibrosis (CF) who are homozygous for the F508del Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) mutation.
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
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject (or his or her legally appointed and authorized representative) will sign and date an informed consent form (ICF).
- •Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
- •Confirmed diagnosis of CF and homozygous for the F508del-CFTR mutation
- •Forced Expiratory Volume in one second/forced vital capacity (FEV1) ≥40% of predicted normal for age, sex, and height
排除标准
- •Presence of moderate or severe hepatic impairment (Child-Pugh Class B or C)
- •Subjects currently receiving invasive mechanical ventilation
- •Known history of alcohol or drug abuse in the past year
- •Clinically significant abnormal laboratory values during screening
- •Pregnant or nursing females
- •Female subjects and female partners of male subjects who plan to become pregnant during Treatment Period or within 90 days following the last dose of study drug
- •History of solid organ or hematological transplantation
- •Ongoing or prior participation in an investigational drug study within 30 days of screening
- •Current use of commercial LUM/IVA combination therapy
研究组 & 干预措施
Arm A: activated smart device alerts and feedback
LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
干预措施: LUM/IVA (Drug)
Arm A: activated smart device alerts and feedback
LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
干预措施: activated smart device (Device)
Arm B: de-activated smart device alerts/feedback features
LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
干预措施: LUM/IVA (Drug)
Arm B: de-activated smart device alerts/feedback features
LUM/IVA: LUM 400 mg q12h/IVA 250 mg q12h through Week 48.
干预措施: de-activated smart device (Device)
结局指标
主要结局
Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment
时间窗: Up to 35 weeks
Percentage adherence was reported in terms of median and full range due to small sample size.
次要结局
- Percentage Adherence to Lumacaftor/Ivacaftor (LUM/IVA) Treatment Through 12 Weeks(Up to Week 12)
- Number of Participants With Greater Than or Equal to (>=) 80 Percent (%) Adherence(Up to Week 12)
- Number of Participants With Greater Than or Equal to (>=) 90 Percent (%) Adherence(Up to Week 12)