NCT03474042已完成2 期
A Phase IIa, Randomized, Double-blind, Placebo-controlled Study to Evaluate GLPG2737 in Orkambi-treated Subjects With Cystic Fibrosis Homozygous for the F508del Mutation
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Galapagos NV
- 入组人数
- 22
- 试验地点
- 9
- 主要终点
- Change from baseline in sweat chloride concentration compared to placebo
研究概览
简要总结
This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d. for 28 days to adult male and female subjects with a confirmed diagnosis of cystic fibrosis homozygous for the F508del CFTR mutation and on stable treatment with Orkambi.
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Male or female subject ≥18 years of age on the day of signing the ICF.
- •A confirmed clinical diagnosis of CF and homozygous for the F508del CFTR mutation.
- •Stable intake of physician prescribed Orkambi (lumacaftor 400 mg/ivacaftor 250 mg b.i.d.) for at least 12 weeks prior to the first study drug administration, and planned continuation of Orkambi for the duration of the study.
- •FEV1 ≥40% of predicted normal for age, gender and height at screening (pre- or postbronchodilator).
- •Sweat chloride concentration ≥60 mmol/L at screening.
排除标准
- •History of serious allergic reaction to any drug as determined by the investigator (e.g., anaphylaxis requiring hospitalization) and/or known sensitivity to any component of the study drug.
- •History of clinically meaningful unstable or uncontrolled chronic disease that makes the subject unsuitable for inclusion in the study in the opinion of the investigator.
- •Unstable pulmonary status or respiratory tract infection (including rhinosinusitis) requiring a change in therapy within 4 weeks prior to the first study drug administration.
- •History of hepatic cirrhosis with portal hypertension (e.g.,signs/symptoms of splenomegaly, esophageal varices, etc.).
- •Abnormal liver function test at screening, defined as aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) and/or alkaline phosphatase and/or gammaglutamyl transferase (GGT) ≥3 x the upper limit of normal (ULN), and/or total bilirubin ≥1.5 x the ULN at screening.
研究组 & 干预措施
GLPG2737
Experimental
GLPG2737 will be provided as capsules for oral use.
干预措施: GLPG2737 (Drug)
Placebo
Placebo Comparator
Placebo will be provided as capsules for oral use.
干预措施: Placebo (Drug)
结局指标
主要结局
Change from baseline in sweat chloride concentration compared to placebo
时间窗: Between day 1 pre-morning dose and Day 28.
To assess Change from baseline in sweat chloride concentration compared to placebo.
次要结局
- Area under the plasma concentration-time curve from time zero until 8 hours (AUC0-8h) post-dose calculated by the linear up - logarithmic down trapezoidal rule (on Day 14)(Between day 1 pre-dose and day 14.)
- Maximum observed plasma concentration of GLPG2737 (Cmax)(Between day 1 pre-dose and day 14.)
- Change versus placebo in the proportion of subjects with adverse events.(Between Day 1 and 3 weeks after the last dose.)
- Change from baseline in sweat chloride concentration.(From baseline (pre-morning dose on Day 1) through 28 days.)
- Change in the respiratory domain of the cystic fibrosis questionnaire-revised (CFQ-R).(From baseline (pre-morning dose on Day 1) through 28 days.)
- Trough plasma concentration observed at the end of the dosing interval (Ctrough).(Between day 1 pre-dose and day 28.)
- Change in percent predicted forced expiratory volume in 1 second (FEV1).(From baseline (pre-morning dose on Day 1) through 28 days.)
研究者
研究点 (9)
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