A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of ELX/TEZ/IVA Combination Therapy in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 64
- 试验地点
- 21
- 主要终点
- Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
The study evaluates the long-term safety, tolerability, efficacy, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in participants with cystic fibrosis (CF).
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completed study drug treatment in parent study (VX18-445-106 Part B, NCT03691779), or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study
排除标准
- •History of study drug intolerance in parent study
- •Other protocol defined Inclusion/Exclusion criteria may apply.
研究组 & 干预措施
ELX/TEZ/IVA
Participants greater than or equal to (≥) 6 years and less than (<) 12 years of age and weighing <30 kilograms (kg) received ELX (elexacaftor) 100 milligram (mg) once daily (qd) /TEZ (tezacaftor) 50 mg qd/IVA (ivacaftor) 75 mg every 12 hours (q12h) and those weighing (≥) 30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/ TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks.
干预措施: ELX/TEZ/IVA (Drug)
ELX/TEZ/IVA
Participants greater than or equal to (≥) 6 years and less than (<) 12 years of age and weighing <30 kilograms (kg) received ELX (elexacaftor) 100 milligram (mg) once daily (qd) /TEZ (tezacaftor) 50 mg qd/IVA (ivacaftor) 75 mg every 12 hours (q12h) and those weighing (≥) 30 kg received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg in the treatment period for up to 192 weeks. Participants ≥12 years of age received ELX 200 mg qd/ TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for up to 192 weeks.
干预措施: IVA (Drug)
结局指标
主要结局
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
时间窗: From Baseline up to Week 196
次要结局
- Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)(From Baseline up to Week 192)
- Absolute Change in Sweat Chloride (SwCl)(From Baseline up to Week 192)
- Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score(From Baseline up to Week 192)
- Absolute Change in Body Mass Index (BMI)(From Baseline up to Week 192)
- Absolute Change in BMI-for-age Z-score(From Baseline up to Week 192)
- Number of Participants With Pulmonary Exacerbations (PEx) for 106/107(From Baseline up to Week 192)
- Number of CF-related Hospitalizations for 106/107(From Baseline up to Week 192)
- Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)(From Baseline up to Week 192)
- Absolute Change in Weight(From Baseline up to Week 192)
- Absolute Change in Weight-for-age Z-score(From Baseline up to Week 192)
- Absolute Change in Height(From Baseline up to Week 192)
- Absolute Change in Height-for-age Z-score(From Baseline up to Week 192)