EUCTR2021-005320-38-DE进行中（未招募）1 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
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 270
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
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 consent.
- •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])11 at the Screening Visit (spirometry measurements must meet American Thoracic Society/European Respiratory Society criteria12 for acceptability and repeatability).
- •6. Subject is able to swallow tablets.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 150
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 120
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
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 x upper limit of normal (ULN)
- •. AST, ALT, gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) = 3 x ULN
- •. 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) 15 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 organisms.
- •. 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 Visit.
- •. 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 of study drug as defined in Table 9-3.
- •10. Subject, or close relative of the subject, is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with
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