A Phase 3b, Open-Label Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Cystic Fibrosis and Advanced Lung Disease, Homozygous for the F508del-CFTR Mutation
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 46
- 主要终点
- Number of Participants With Treatment Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)
研究概览
简要总结
The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Homozygous for the F508del-CFTR mutation; historical genotype must be documented in the participant's source documents.
- •Percent predicted FEV1 <40 of adjusted for age, sex, and height at Screening
排除标准
- •Participant currently receiving invasive mechanical ventilation.
- •History of any comorbidity 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 participant
- •Any clinically significant laboratory abnormalities at screening that would interfere with the study assessments or pose an undue risk for the subject
- •A 12-lead electrocardiograms (ECG) demonstrating QTcF >450 msec at Screening
- •History of solid organ or hematological transplantation
- •History of alcohol or drug abuse in the past year
- •Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of screening.
- •Use of strong inhibitors, moderate inducers, or strong inducers of CYP3A
- •Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at Screening and Day
- •Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements
- •Use of beta blockers or the equivalent at Screening.
研究组 & 干预措施
Lumacaftor/Ivacaftor combination
Lumacaftor 400 milligram (mg) and ivacaftor 250 mg combination tablet orally twice daily for 24 weeks.
干预措施: Lumacaftor (Drug)
Lumacaftor/Ivacaftor combination
Lumacaftor 400 milligram (mg) and ivacaftor 250 mg combination tablet orally twice daily for 24 weeks.
干预措施: Ivacaftor (Drug)
结局指标
主要结局
Number of Participants With Treatment Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)
时间窗: Day 1 up to Week 28
AE: any untoward medical occurrence in a participant during the study; event does not necessarily have a causal relationship with treatment. This includes any newly occurring event/previous condition that has increased in severity/frequency after informed consent form is signed. AE includes serious as well as non-serious AEs. SAE (subset of AE): medical event, which falls into any of the following categories, regardless of its relationship to study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. TEAEs: AEs that started/ worsened on/after the start of study drug through the Safety Follow up Visit (4 weeks after the last dose of study drug). Results were reported as planned, as a combined single LUM/IVA arm irrespective of permitted dose modification.
次要结局
- Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24(Baseline, Up to Week 24)
- Absolute Change From Baseline in Sweat Chloride at Average of Day 15 and Week 4(Baseline, Day 15 and Week 4)
- Number of Hospitalizations(Baseline through Week 24)
- Absolute Change From Baseline in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score Through Week 24(Baseline, Through Week 24)
- Absolute Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24(Baseline, Up to Week 24)
- Duration For Which Participants Received Intravenous (IV) Antibiotics(Baseline through Week 24)