EUCTR2012-000386-20-GB进行中（未招募）1 期
A Phase 3, 2-Arm, Roll-Over Study to Evaluate the Long-term Safety and Pharmacodynamics of Ivacaftor Treatment in Pediatric Subjects With Cystic Fibrosis and a CFTR Gating Mutation
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
- 33
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
入选标准
- •Inclusion Criteria: Ivacaftor Arm
- •1.Completed the last study visit of the treatment period of the previous study
- •2.Hematology, serum chemistry, and vital signs results on Day 1 (most recent visit from the previous study on which these data are available) with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator
- •3. 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 assents to participation in the study to the degree the subject can assent, and that the subject will comply with and is likely to complete the study as planned
- •4. Parent or legal guardian must sign the ICF and corresponding assent must be obtained from the subject, as applicable
- •Inclusion Criterion: Observational Arm
- •Subjects who completed their assigned study drug treatment in the previous study and elected not to enroll in the ivacaftor arm and subjects who prematurely discontinued treatment in the previous study and received at least 1 dose of study drug treatment in the previous study will be eligible for enrollment in the observational arm.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 35
- •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
- •Exclusion Criteria: Ivacaftor Arm
- •1.Subjects who prematurely discontinued from the previous study
- •2.History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject
- •3.Subjects with a history of study treatment intolerance as observed in their previous study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
- •4.Subjects receiving commercially-available ivacaftor treatment
- •5.Subject was unable to complete an adequate slit-lamp examination at the last ophthalmologic examination in the previous study
- •Exclusion Criterion: Observational Arm
- •1.Subjects receiving ivacaftor treatment will not be eligible for enrollment in the observational arm.
研究者
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