A Phase I, Randomized, Placebo-Controlled, Study Designed to Assess the Safety, Tolerability, and Pharmacokinetics of PTI-428 in Subjects With Cystic Fibrosis.
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 2
- 主要终点
- Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs
研究概览
简要总结
The trial will consist of a single treatment group enrolling adult subjects with CF on background therapy with KALYDECO®. Approximately 16 subjects will be enrolled.
详细描述
The single treatment group will enroll adult subjects with CF currently on stable KALYDECO® background therapy for a minimum of 3 months at the time of randomization. Subjects will be randomized to either PTI-428 or placebo. Each dose will be administered once daily (QD) for a total of 14 Days. The subjects will continue treatment with KALYDECO® throughout the study.
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
- •Confirmed diagnosis of CF.
- •Stable on Kalydeco dosing for both label indication and per label dosing for a minimum of 3 months at the time of randomization
- •Forced expiratory volume in 1 second (FEV1) 40-90% predicted.
- •Non-smoker and non-tobacco user for a minimum of 30 days prior to screening and for the duration of the study.
排除标准
- •Participation in another clinical trial or treatment with an investigational agent within 30 days or 5 half-lives, whichever is longer, prior to Study Day
- •History of cancer within the past five years (excluding cervical CIS with curative therapy for at least one year prior to screening and non-melanoma skin cancer).
- •History of organ transplantation.
- •Any sinopulmonary infection or CF exacerbation requiring a change or addition of medication (including antibiotics) within 1 month of Study Day 1 or any other clinically significant infection as determined by the investigator within 1 month of Day
- •History of alcohol or drug abuse or dependence within 12 months of screening as determined by the Investigator.
- •Male and female of child-bearing potential, unless they are using highly effective methods of contraception during participation in the clinical study and for 4 weeks after termination from study.
- •Pregnant or nursing women.
研究组 & 干预措施
PTI-428
Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.
干预措施: PTI-428 (Drug)
Placebo
Subjects will receive once daily dosing of PTI-428 or placebo for 14 days.
干预措施: Placebo (Drug)
结局指标
主要结局
Safety and tolerability as assessed by adverse events, safety labs, electrocardiograms (ECGs), physical examinations, and vital signs
时间窗: Baseline through Day 21
次要结局
- Cmax of multiple oral doses(Baseline through Day 14)
- t1/2 of multiple oral doses(Baseline through Day 14)
- Tmax of multiple oral doses(Baseline through Day 14)
- AUC0-t of multiple oral doses(Baseline through Day 14)