EUCTR2020-002251-38-DE进行中（未招募）1 期
A Phase 3 Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Through 5 Years of Age
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 70
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’s legally appointed and authorized representative will sign and date an informed consent form (ICF).
- •2. Subjects (males and females), 2 through 5 years of age, inclusive, on the date of informed consent (and assent, as applicable).
- •3. In Part A, subjects must weigh =14 kg at Day 1. In Part B, subjects must weigh =10 kg at the Screening Visit.
- •4. Confirmed diagnosis of CF as determined by the investigator.
- •5. In Part A, subjects who are homozygous for F508del (F/F genotype) or heterozygous for F508del and an MF mutation that is not responsive to IVA and TEZ/IVA (F/MF genotypes). In Part B, subjects who have at least 1 F508del mutation in the CFTR gene or an ELX/TEZ/IVA-responsive CFTR mutation.
- •Genotype should be confirmed at the Screening Visit. This assessment does not need to be repeated for confirmed subjects in Part A who wish to participate in Part B.
- •If the screening CFTR genotype result is not received before the first dose of study drug, a previous CFTR genotype laboratory report may be used to establish eligibility.
- •Subjects who have been enrolled and whose screening genotype does not confirm study eligibility must be discontinued from the study.
- •6. Subjects with stable CF disease at the start of the Treatment Period as deemed by the investigator.
- •7. Subjects who are willing to remain on a stable CF medication regimen (other than CFTR modulators) through Day 15 (Part A) or through Week 24 (Part B) or, if applicable, through the Safety Follow-up Visit.
- •8. As judged by the investigator, the 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
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 70
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •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, 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. This includes, but is not limited to, the following:
- •Clinically significant cirrhosis with or without portal hypertension
- •Solid organ or hematological transplantation
- •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, aspartate transaminase (AST), or alanine transaminase (ALT) =2 × upper limit of normal (ULN)
- •Alkaline phosphatase (ALP) or gamma-glutamyl transferase (GGT) =3 × ULN
- •Abnormal renal function defined as glomerular filtration rate =45 mL/min/1.73 m2 (calculated by the Counahan-Barratt equation)8
- •4. An acute upper or lower respiratory infection, PEx, or changes in therapy (including antibiotics) for pulmonary 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 Mycobacterium 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.
- •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 (including studies investigating ELX with or without coadministration with other study drugs) 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.
- •Note: Ongoing participation in a noninterventional study (including observational studies) is permitted.
- •8. Use of restricted medication within specified duration before the first dose of study drug as defined in Table 9-2.
- •9. The subject or a close relative of the subject is the investigator or a subinvestigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study
- •10. Part B only: Elevated serum ALT or AST =3 × ULN or total bilirubin = 2 × ULN in the previous year.
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