2024-515606-90-00招募中3 期
A Phase 3 Open-label Study Evaluating the Long term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Years and Older
适应症
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 3
- 试验地点
- 2
- 主要终点
- Safety and tolerability of ELX/TEZ/IVA based on adverse events (AEs), clinical laboratory values, standard 12-lead ECGs, vital signs, and pulse oximetry
研究概览
简要总结
To evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in cystic fibrosis (CF) subjects 2 years of age and older
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 否
入选标准
- •Part A: Subject’s legal representative or guardian will sign and date an informed consent form (ICF).
- •Part B: Willing to remain on a stable CF treatment regimen (as defined in Section 9.5) through completion of study participation
- •Part A: As judged by the investigator, the legal representative or guardian must be able to understand protocol requirements, restrictions, and instructions and the legal representative or guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned
- •Part A: 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, or • Had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study.
- •Part A: Willing to remain on a stable CF treatment regimen (as defined in Section 9.5) through completion of study participation.
- •Part B: Subject’s legal representative or guardian will sign and date an ICF.
- •Part B: As judged by the investigator, the legal representative or guardian must be able to understand protocol requirements, restrictions, and instructions and the legal representative or guardian should be able to ensure that the subject will comply with and is likely to complete the study as planned.
- •Part B: Did not withdraw consent in Part A.
- •Part B: Meets at least 1 of the following criteria: • Completed study drug treatment in Part A, or • Had study drug interruption(s) in Part A, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of Part A.
排除标准
- •Part A: 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. This includes, but is not limited to, the following: • Clinically significant liver cirrhosis with or without portal hypertension • Solid organ or hematological transplantation • Cancer
- •Part B: History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study or Part A, as deemed by the investigator.
- •Part B: Use of restricted medication, as defined in Table 9-1, unless subject is on a study drug interruption at the time of rollover.
- •Part B: The subject or a close relative of the subject is the investigator or a sub-investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site.
- •Part A: 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).
- •Part A: 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.
- •Part A: History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study as deemed by the investigator.
- •Part A: Use of restricted medication, as defined in Table 9-1, unless subject is on a study drug interruption at the time of rollover.
- •Part A: The subject or a close relative of the subject is the investigator or a sub-investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site.
- •Part B: History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug(s) to the subject.
- •Part B: History of drug intolerance in the parent study or Part A that would pose an additional risk to the subject in the opinion of the investigator (e.g., subjects with a history of allergy or hypersensitivity to the study drug).
- •Part B: Current participation in an investigational drug trial other than the parent study or the current 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
结局指标
主要结局
Safety and tolerability of ELX/TEZ/IVA based on adverse events (AEs), clinical laboratory values, standard 12-lead ECGs, vital signs, and pulse oximetry
Safety and tolerability of ELX/TEZ/IVA based on adverse events (AEs), clinical laboratory values, standard 12-lead ECGs, vital signs, and pulse oximetry
次要结局
- Absolute change in sweat chloride (SwCl)
- Absolute change in lung clearance index (LCI)2.5
研究者
Clinical Trials and Medical Info
Scientific
Vertex Pharmaceuticals Inc.
研究点 (2)
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