A Phase IIa, Open-label Study of Multiple Doses of GLPG1837 in Subjects With Cystic Fibrosis and the G551D Mutation
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Galapagos NV
- 入组人数
- 26
- 试验地点
- 16
- 主要终点
- Changes in vital signs - composite outcome measure
研究概览
简要总结
32 cystic fibrosis patients with the G551D mutation will be treated for 4 weeks, consisting of three consecutive treatment periods: two 1-week periods followed by one 2-week period, evaluating one dose of GLPG1837 each. After the treatment period, there is a 7-10 days follow-up period.
During the course of the study, subjects will be examined for any side effects that may occur (safety and tolerability).
Changes in sweat chloride will be assessed as biomarker from baseline onwards, and changes in pulmonary function (efficacy) will be explored throughout the study. The amount of GLPG1837 present in the blood (pharmacokinetics) will also be determined.
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
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female subjects ≥ 18 years of age, with a confirmed diagnosis of cystic fibrosis
- •Subjects with gating G551D CFTR mutation on at least one allele in the CFTR gene
- •Subjects currently receiving treatment with ivacaftor on a stable regimen or not on a treatment regimen with ivacaftor, for at least 2 weeks prior to screening
- •Weight ≥ 40.0 kg
- •Subjects on stable concomitant treatment regimen for at least 4 weeks prior to baseline (excluding ivacaftor)
- •Pre- or post-bronchodilator FEV1 ≥ 40% of predicted normal
- •Subject will have to use highly effective contraceptive methods
排除标准
- •On an ivacaftor-containing treatment regimen and unable or unwilling to discontinue ivacaftor for the washout and treatment periods of the study
- •Concomitant use of antifungal drugs within 4 weeks of baseline
- •A history of a clinically meaningful unstable or uncontrolled chronic disease
- •Liver cirrhosis and portal hypertension
- •Any significant change in the medical regimen for pulmonary health within 4 weeks of baseline
- •Unstable pulmonary status or respiratory tract infection or changes in therapy for pulmonary disease within 4 weeks of baseline
- •Abnormal liver function
- •Clinically significant abnormalities on ECG
- •History of malignancy, solid organ/haematological transplantation
- •Abnormal renal function
- •Participation in another experimental therapy study within 30 days or 5 times halflife
研究组 & 干预措施
GLPG1837 dose 1, GLPG1837 dose 2, GLPG1837 dose 3
GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks
干预措施: GLPG1837 dose 1 (Drug)
GLPG1837 dose 1, GLPG1837 dose 2, GLPG1837 dose 3
GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks
干预措施: GLPG1837 dose 2 (Drug)
GLPG1837 dose 1, GLPG1837 dose 2, GLPG1837 dose 3
GLPG1837 twice daily oral dosing - morning and evening, for 4 weeks
干预措施: GLPG1837 dose 3 (Drug)
结局指标
主要结局
Changes in vital signs - composite outcome measure
时间窗: Up to 9 weeks
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal vital signs as measured by temperature, blood pressure, heart rate and respiratory rate, at every visit
Changes in adverse events
时间窗: Up to 9 weeks
To evaluate the safety and tolerability of GLPG1837 in terms of adverse events at every visit
Changes in laboratory parameters
时间窗: Up to 7 weeks
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal laboratory parameters at every visit
Changes in electrocardiogram
时间窗: Up to 7 weeks
To evaluate the safety and tolerability of GLPG1837 in terms of abnormal electrocardiogram at every visit
Changes in physical examination - composite outcome measure
时间窗: Up to 9 weeks
To evaluate the safety and tolerability of GLPG1837 in terms of abnormalities during physical examination at every visit
次要结局
- Changes in sweat chloride concentration(Up to 9 weeks)
- Changes in pulmonary function (forced expiratory volume in 1 second, FEV1) assessed by spirometry(Up to 9 weeks)
- Plasma levels of GLPG1837: Cmax, the maximum observed plasma concentration(Up to 3 weeks)
- Plasma levels of GLPG1837: tmax, the time of occurrence of Cmax(Up to 3 weeks)
- Plasma levels of GLPG1837: AUC, the area under the plasma concentration-time curve(Up to 3 weeks)