A Phase 3b, 2-part, Randomized, Double-blind, Placebo-controlled Crossover Study With a Long term Open-label Period to Investigate Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation

Vertex Pharmaceuticals Incorporated0 个研究点目标入组 14 人开始时间: 2015年8月24日最近更新: 2019年6月30日

适应症

试验速览

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•1. Male or female with confirmed diagnosis of CF, defined as:
•o a sweat chloride value =60 mmol/L by quantitative pilocarpine iontophoresis
•2 CF causing mutations (all as documented in the subject's medical record)
•o Clinical manifestations of CF
•2. Must have 1 of the following CFTR gating mutations on at least 1 allele: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D.
•3. Subjects will be between the ages of 3 and 5 years, inclusive, at Screening and Day 1
•4. Weight =8 kg to <25 kg at Screening and Day 1
•5. Hematology, serum chemistry, and coagulation at Screening with no clinically significant abnormalities or concomitant diagnosis that would interfere with the LCI and CT scan study assessments, as judged by the investigator
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 50
•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

•1. History of any illness or condition that, in the opinion of the investigator, might confound the results of the study, impact use of the LCI or CT scan as assessments or pose an additional risk in administering study drug to the subject
•2. An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
•3. Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for the subject (in the opinion of the investigator)
•4. Abnormal liver function, at Screening, defined as 3 × upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), and total bilirubin
•5. History of solid organ or hematological transplantation
•6. Any clinically significant non-CF-related illness within 2 weeks before Day 1. Illness is defined as an acute (serious or nonserious) condition (e.g., gastroenteritis)
•7. Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A within 2 weeks before Day 1
•8. Participation in a clinical study involving administration of either an investigational or a marketed drug within 30 days or 5 terminal half-lives (whichever is longer or as determined by the local requirements) before Screening

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