NCT02589236已完成2 期
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 to Evaluate Efficacy and Safety in Patients With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation Treated With Lumacaftor/Ivacaftor
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 138
- 试验地点
- 46
- 主要终点
- Absolute change from baseline in percent predicted FEV1 (ppFEV1)
研究概览
简要总结
This will be a double-blind, randomized, placebo-controlled, parallel group study. The purpose of this study is to investigate the efficacy and safety of Cavosonstat (N91115) in adult patients with CF who are homozygous for the F508del-CFTR mutation and being treated with lumacaftor/ivacaftor (Orkambi™).
详细描述
Primary Objective:
- Assess the efficacy of N91115 at 12 weeks when added to preexisting treatment with lumacaftor/ivacaftor in adult patients with CF who are homozygous for the F508del-CFTR mutation
Secondary Objectives:
- Assess the effect of N91115 added to lumacaftor/ivacaftor on safety
- Assess the effect of lumacaftor/ivacaftor added to N91115 on the pharmacokinetics of N91115, lumacaftor, and ivacaftor
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must have been treated with lumacaftor/ivacaftor for at least 8 weeks prior to Day 1 (start of dosing)
- •A history of Sweat Chloride (SC) ≥ 60 mEq/L by quantitative pilocarpine iontophoresis test (QPIT) (either before or after starting lumacaftor/ivacaftor treatment)
- •Body weight ≥ 40 kg
- •ppFEV1 40 - 85 % predicted (inclusive) at screening
- •Oxygen saturation ≥ 90% breathing ambient air at screening
排除标准
- •Any acute infection that requires treatment or hospitalization within 2 weeks of Study Day 1
- •Colonization with organisms associated with more rapid decline in pulmonary status, such as Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus
- •Any change in the regimen for chronic therapies for CF lung disease (e.g., Pulmozyme®, hypertonic saline, Azithromycin, TOBI®, Cayston®) within 4 weeks of Study Day 1
- •Are pregnant, planning a pregnancy, or breast-feeding at screening
- •Blood hemoglobin < 10 g/dL at screening
- •Serum albumin < 2.5 g/dL at screening
- •Abnormal liver function defined as ≥ 3 x upper limit of normal (ULN)
- •History of abnormal renal function within 3 months of screening
- •History of ventricular tachycardia or other clinically significant ventricular arrhythmias
- •History, including the screening assessment, of prolonged QT and/or QTcF (Fridericia's correction) interval
- •History of solid organ or hematological transplantation
- •History of alcohol abuse or drug abuse
- •Ongoing participation in another therapeutic clinical trial
- •Use of continuous (24 hr/day) or nocturnal supplemental oxygen
研究组 & 干预措施
Placebo
Placebo Comparator
Placebo Capsule
干预措施: Placebo (Drug)
Cavosonstat (N91115) 200 mg
Experimental
Cavosonstat (N91115) 200 mg twice daily (BID)
干预措施: Cavosonstat (Drug)
Cavosonstat (N91115) 400 mg
Experimental
Cavosonstat (N91115) 400 mg BID
干预措施: Cavosonstat (Drug)
结局指标
主要结局
Absolute change from baseline in percent predicted FEV1 (ppFEV1)
时间窗: From baseline to 12 weeks
Forced Expiratory Volume in one second (FEV1) from before study (Baseline) to after 12 weeks of N91115 treatment
次要结局
- Absolute change from baseline in Cystic Fibrosis Questionnaire -Revised CFQ-R (respiratory symptom scale)(baseline to 16 weeks)
- Absolute change from baseline in Patient Global Impression of Change (PGIC)(baseline to 12 weeks)
- Relative change from baseline in ppFEV1(baseline to 12 weeks)
- Absolute change from baseline in sweat chloride(baseline to 12 weeks)
- Absolute change from baseline in body mass index (BMI)(baseline to 12 weeks)
- Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability])(baseline to 16 weeks)
- Pharmacokinetic Measurements of Maximum Plasma Concentration [Cmax], of N91115, lumacaftor, and ivacaftor(baseline to 12 weeks)
- Pharmacokinetic Measurements of Area Under the Curve (AUC) for N91115, Ivacaftor and lumacaftor(baseline to 12 weeks)
研究者
研究点 (46)
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