2024-515637-14-00招募中3 期
A Phase 3 Open-label Study Evaluating the Longterm Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes
适应症
干预措施
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 295
- 试验地点
- 77
- 主要终点
- Parts A and B: Safety and tolerability based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry
研究概览
简要总结
Part A and B: To evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA)
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Subject (or the subject’s legally appointed and authorized representative) will sign and date an informed consent form (ICF) and, when appropriate, an assent form.
- •Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines (as applicable), and other study procedures. • For subjects <18 years of age: as judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions and the parent or legal guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned.
- •Did not withdraw consent from the parent study.
- •Part A: Meets at least 1 of the following criteria: • Completed study drug treatment in the parent study. • Had study drug interruption(s) in the parent study, but completed study visits up to the last scheduled visit of the Treatment Period of the parent study. Part B: Meets at least 1 of the following criteria: • Completed study drug treatment in Part A. • Had study drug interruption(s) in Part A, but completed study visits up to the last scheduled visit of the Treatment Period of Part A.
- •Willing to remain on a stable CF treatment regimen (other than CFTR modulators, as defined in Section 9.5) through completion of study participation.
排除标准
- •History of any illness or any clinical condition that might confound the results of the study or pose an additional risk in administering study drug(s) to the subject.
- •History of drug intolerance in the parent study that would pose an additional risk to the subject. (e.g., subjects with a history of allergy or hypersensitivity to the study drug).
- •Pregnant and nursing females. Females of childbearing potential (Section 11.5.6.1) must have a negative pregnancy test at the Day 1 Visit (in Part A and Part B) before receiving the first dose of study drug.
- •Current participation in an investigational drug trial (other than the parent study). Participation in a noninterventional study (including observational studies, registry studies, and studies requiring blood collections without administration of study drug) and screening for another Vertex study is permitted.
研究组 & 干预措施
Kalydeco 75 mg film-coated tablets
Test
干预措施: Kalydeco 75 mg film-coated tablets (Drug)
Kaftrio 37.5 mg/25 mg/50 mg film-coated tablets
Test
干预措施: Kaftrio 37.5 mg/25 mg/50 mg film-coated tablets (Drug)
VX-445/VX-661/VX-770 fixed-dose combination tablet
Test
干预措施: VX-445/VX-661/VX-770 fixed-dose combination tablet (Drug)
VX-770 Film-coated tablet
Test
干预措施: VX-770 Film-coated tablet (Drug)
Kaftrio 75 mg/50 mg/100 mg film-coated tablets
Test
干预措施: Kaftrio 75 mg/50 mg/100 mg film-coated tablets (Drug)
Kalydeco 150 mg film-coated tablets
Test
干预措施: Kalydeco 150 mg film-coated tablets (Drug)
VX-445/VX-661/VX-770 film-coated fixed-dose combination tablet
Test
干预措施: VX-445/VX-661/VX-770 film-coated fixed-dose combination tablet (Drug)
结局指标
主要结局
Parts A and B: Safety and tolerability based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry
Parts A and B: Safety and tolerability based on adverse events (AEs), clinical laboratory values, ECGs, vital signs, and pulse oximetry
次要结局
- Number of pulmonary exacerbations (PEx)
- Absolute change from baseline in weight
- Part A Only: Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1)
- Absolute change from baseline in sweat chloride (SwCl)
- Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain (RD) score
- Absolute change from baseline in body mass index (BMI)
研究者
Clinical Trials and Medical Info
Scientific
Vertex Pharmaceuticals Inc.
研究点 (77)
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