A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect of PTI-428 in Subjects With Cystic Fibrosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 40
- 试验地点
- 26
- 主要终点
- Number of subjects with treatment-emergent adverse events (TEAEs)
研究概览
简要总结
The study population is comprised of adult subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation and are currently receiving background treatment with tezacaftor/ivacaftor for a minimum of 1 month prior to Day 1. The planned sample size is approximately 40 subjects. 20 subjects will be assigned to PTI-428 dose level 1 or placebo and 20 subjects will be assigned to PTI-428 dose level 2 or placebo. At each dose level, subjects will be randomized at a 3:1 randomization ratio. Subjects will receive once daily oral doses of PTI-428 or placebo for 28 days, while the subjects continue to receive background treatment with tezacaftor/ivacaftor per product label. The study drug administration period will be followed by a 14-day safety follow-up period.
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 with the F508del/F508del genotype on record
- •On tezacaftor/ivacaftor dosing for both label indication and per label dosing for a minimum of 1 month on Day 1
- •Forced expiratory volume in 1 second (FEV1) 40-90% predicted, inclusive
- •Clinically stable with no significant changes in health status within 14 days of Day 1
- •Non-smoker and non-tobacco user for a minimum of 28 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 28 days or 5 half-lives, whichever is longer, prior to Study Day 1
- •History of cancer within the past 5 years (excluding cervical cancer in situ with curative therapy for at least one year prior to screening and non-melanoma skin cancer)
- •History of organ transplantation
- •Hospitalization, sinopulmonary infection, CF exacerbation, or other clinically significant infection or illness (as determined by the investigator) requiring an increase or addition of medication, such as antibiotics or corticosteroids, within 14 days of Day 1
- •Initiation of any new chronic therapy (e.g., ibuprofen, hypertonic saline, azithromycin, Pulmozyme®, Cayston®, TOBI®)) or any change in chronic therapy (excluding pancreatic enzyme replacement therapy) within 28 days prior to Day 1
- •History or current evidence of alcohol or drug abuse or dependence within 12 months of screening as determined by the investigator
- •Pregnant or nursing women
研究组 & 干预措施
PTI-428 dose level 1
干预措施: PTI-428 (Drug)
PTI-428 dose level 2
干预措施: PTI-428 (Drug)
Placebo PTI-428
干预措施: Placebo (Drug)
结局指标
主要结局
Number of subjects with treatment-emergent adverse events (TEAEs)
时间窗: Baseline through Day 42
Safety and tolerability will be assessed by adverse events (AEs), safety labs, electrocardiograms (ECGs), physical examinations and vital signs.
次要结局
- Time of Cmax (Tmax)(28 days)
- Change in FEV1 over time(Baseline through Day 42)
- Change in sweat chloride over time(Baseline through Day 42)
- Maximum plasma concentration (Cmax)(28 days)
- Area under the concentration time curve from time 0 to time of last measurable concentration (AUC0-t)(28 days)