NCT03125395已完成3 期
A Phase 3, Rollover Study to Evaluate the Safety of Long-term Treatment With Lumacaftor/Ivacaftor Combination Therapy in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 57
- 试验地点
- 20
- 主要终点
- Safety as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
研究概览
简要总结
A Rollover Safety Study of Lumacaftor/Ivacaftor in Subjects Aged 2 Years and Older With Cystic Fibrosis, Homozygous for F508del.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subjects entering the Treatment Cohort must meet the following criteria:
- •Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study VX15-809-115 Part B (Study 115B, NCT02797132)
- •Willing to remain on a stable CF medication regimen through the Safety Follow-up Visit
- •Subjects entering the Observational Cohort must meet 1 of the following criteria:
- •Completed 24 weeks of LUM/IVA treatment and the Safety Follow-up Visit in Study 115B, but do not want to enroll in the Treatment Cohort.
- •Received at least 4 weeks of LUM/IVA treatment and completed visits up to Week 24 and the Safety Follow-up Visit, if required, of Study 115B but are not taking LUM/IVA at the end of the Study 115B Treatment Period (i.e., Week 24) because of a drug interruption and either did not receive Vertex approval to enroll in the Treatment Cohort or do not want to enroll in the Treatment Cohort.
- •Permanently discontinued LUM/IVA in Study 115B after receiving at least 4 weeks of treatment and remained in the study from the time of treatment discontinuation through the Week 24 Visit and Safety Follow-up Visit, if required.
- •Exclusion Criteria (Treatment Cohort Only):
- •Prematurely discontinued LUM/IVA treatment in Study 115B.
- •History of any comorbidity or laboratory abnormality that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering LUM/IVA to the subject
- •History of drug intolerance or other serious reactions to LUM/IVA in Study 115B that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor.
- •Subjects with a history of allergy or hypersensitivity to LUM/IVA.
- •Liver function test (LFT) abnormality meeting criteria for LUM/IVA treatment interruption at the completion of Study 115B, for which no convincing alternative etiology is identified.
- •QTc value at the completion of Study 115B that would pose an additional risk to the subject in the opinion of investigator, and which should be discussed with the Vertex medical monitor
- •History of poor compliance with LUM/IVA and/or procedures in Study 115B as deemed by the investigator.
- •Participation in an investigational drug trial (including studies investigating LUM and/or IVA) other than Study 115B.
排除标准
- 未提供
研究组 & 干预措施
LUM/IVA
Experimental
LUM/IVA granules or tablets were administered orally every 12 hours (Participants aged 2 through 5 years received LUM 100 mg/IVA 125 mg granules or LUM 150 mg/IVA 188 mg granules based on body weight. Participants ≥6 years of age were to receive LUM 200 mg/IVA 250 mg tablets). Doses were adjusted upward for changes in weight and age.
干预措施: LUM/IVA (Drug)
结局指标
主要结局
Safety as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
时间窗: Day 1 up to Week 98
次要结局
- Absolute Change From Baseline in Stature (Height)(From Parent Study 115B Baseline at Week 96)
- Absolute Change in Stature-for-age Z-score(From Parent Study 115B Baseline at Week 96)
- Absolute Change in Sweat Chloride(From Parent Study 115B Baseline at Week 96)
- Absolute Change in Immunoreactive Trypsinogen (IRT) Serum Levels(From Parent Study 115B Baseline at Week 96)
- Absolute Change in Weight-for-age Z-score(From Parent Study 115B Baseline at Week 96)
- Absolute Change in Body Mass Index (BMI)(From Parent Study 115B Baseline at Week 96)
- Absolute Change in Weight(From Parent Study 115B Baseline at Week 96)
- Absolute Change in BMI-for-age Z-score(From Parent Study 115B Baseline at Week 96)
- Number of Participants With Microbiology Culture Status (Positive or Negative)(Week 96)
- Absolute Change in Lung Clearance Index (LCI) 2.5(From Parent Study 115B Baseline at Week 96)
- Time-to-first Pulmonary Exacerbation(From Parent Study 115B Baseline through Week 96)
- Number of Pulmonary Exacerbations (PEx)(From Parent Study 115B Baseline through Week 96)
- Absolute Change in Lung Clearance Index (LCI) 5.0(From Parent Study 115B Baseline at Week 96)
- Number of Cystic Fibrosis (CF) Related Hospitalizations(From Parent Study 115B Baseline through Week 96)
- Absolute Change in Fecal Elastase-1 (FE-1) Levels(From Parent Study 115B Baseline at Week 96)
研究者
研究点 (20)
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