NL-OMON51518已完成3 期
A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR Mutation - Evaluation of ELX/TEZ/IVA in Subjects Without F508del Mutation
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 15
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 64（—）
入选标准
- •1. 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.
- •2. 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.
- •3. Subjects (male or female) 6 years of age and older on the date of informed
- •4. Subjects has an eligible ELX/TEZ/IVA-responsive CFTR mutation listed in
- •Table 15-1 and none of the exclusionary mutations in Table 15-2.
- •5. Subject has stable CF disease, as deemed by the investigator, before
- •randomization.
- •a. Forced expiratory volume in 1 second (FEV1) value >=40% and <=100% of
- •predicted mean for age, sex, and height (equations of the Global Lung Function
- •Initiative [GLI]) at the Screening Visit (spirometry measurements must meet
- •American Thoracic Society/European Respiratory Society criteria for
- •acceptability and repeatability).
- •6. Subject is able to swallow tablets.
排除标准
- •1. 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 may include, but is not limited to:
- •History of allergy, intolerance, or hypersensitivity to any component of the
- •investigational drug product (ELX/TEZ/IVA tablets and IVA tablets) or placebo,
- •including excipients
- •Clinically significant liver cirrhosis with or without portal hypertension
- •Solid organ or hematological transplantation
- •Alcohol or drug abuse in the past year, including, but not limited to,
- •cannabis, cocaine, and opiates, as deemed by the investigator
- •Cancer, except for squamous cell skin cancer, basal cell skin cancer, and Stage
- •0 cervical carcinoma in situ (all 3 with no recurrence for the last 5 years).
- •2. Any clinically significant laboratory abnormalities at the Screening Visit
- •that would interfere with the study assessments or pose an undue risk for the
- •subject (as deemed by the investigator).
- •3. Any of the following abnormal laboratory values at screening:
- •. Hemoglobin <10 g/dL
- •. Total bilirubin >=2 × upper limit of normal (ULN)
- •. AST, ALT, gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) >= 3
- •. Abnormal renal function defined as glomerular filtration rate <=50 mL/min/1.73
- •m2 (calculated by the Modification of Diet in Renal Disease Study Equation) for
- •subjects >=18 years of age and <=45 mL/min/1.73 m2 (calculated by the
- •Counahan-Barratt equation) for subjects < 18 years of age.
- •4. An acute upper or lower respiratory infection, pulmonary exacerbation, or
- •changes in therapy (including antibiotics) for sinopulmonary disease within 28
- •days before Day 1 (first dose of study drug).
- •5. Lung infection with organisms associated with a more rapid decline in
- •pulmonary status (including, but not limited to, Burkholderia cenocepacia,
- •Burkholderia dolosa, and Mycobaterium abscessus). For subjects who have had a
- •history of a positive culture, the investigator will apply the following
- •criteria to establish whether the subject is free of infection with such
- •. The subject has not had a respiratory tract culture positive for these
- •organisms within the 12 months before the date of informed consent, and
- •. The subject has had at least 2 respiratory tract cultures negative for such
- •organisms within the 12 months before the date of informed consent, with the
- •first and last of these separated by at least 3 months, and the most recent one
- •within the 6 months before the date of informed consent.
- •6. An acute illness not related to CF (e.g., gastroenteritis) within 14 days
- •before the first dose of study drug (Day 1).
- •7. Ongoing or prior participation in an investigational drug study within 28
- •days of the Screening Visit.
- •. A washout period of 5 terminal half-lives of the previous investigational
- •study drug, or 28 days, whichever is longer, must elapse before the Screening
- •. The duration of the elapsed time may be longer if required by local
- •regulations.
- •8. Pregnant and breast-feeding females. Female subjects of childbearing
- •potential (Section 11.5.6.1) must have a negative pregnancy test at the
- •Screening Visit and the Day 1 Visit.
- •9. Use of restricted medication within specified duration before the first dose
研究者
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