A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of P-1037 Solution for Inhalation in Patients With Cystic Fibrosis (CF)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 142
- 试验地点
- 33
- 主要终点
- Safety (number of adverse events of P-1037 in treatment groups)
研究概览
简要总结
The goal of the study is to evaluate the safety and tolerability of P-1037 and to determine whether the combination of P-1037 with hypertonic saline or P-1037 alone has a greater effect on lung function in patients with CF than placebo (0.17% saline).
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
- 12 Years 至 80 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female, 12 years of age or older.
- •Diagnosis of cystic fibrosis as determined by the 1997 CF Consensus criteria (NIH Consensus Statement, 1997)
- •Non-smoker
- •FEV1 at Screening Visit 1 between 40% and 90%
- •Stable regimen of CF medications and chest physiotherapy for the 28 days prior to Screening. Must be willing to discontinue use of hypertonic saline for the duration of the study.
- •Clinically stable for at least 2 weeks
- •All females of child-bearing potential must have a negative serum pregnancy test and if sexually active must agree to practice a highly effective form of contraception throughout the study and for 28 days after the last dose of study medication.
排除标准
- •History of any organ transplantation or any significant disease or disorder
- •Use of diuretics (including amiloride) or renin-angiotensin antihypertensive drugs or trimethoprim in the 28 days prior to Screening
- •History of significant intolerance to inhaled hypertonic saline, as determined by the Investigator
- •Known hypersensitivity to the study drug or amiloride
- •Any clinically significant laboratory abnormalities at Screening Visit 1 as judged by the investigator, or any of the following:
- •Potassium ≥ 5 milliequivalent per Liter (mEq/L)
- •Abnormal renal function
- •Abnormal liver function, defined as ≥ 3 x upper limit of normal (ULN)
- •Hemoglobin level < 10.0 g/dL
- •Female who is pregnant or lactating
- •History of sputum or throat swab culture yielding Burkholderia species or Mycobacterium abscessus within 2 years of screening
- •Has previously participated in an investigational trial involving administration of any investigational compound or use of an investigational device with 28 days prior to Screening
- •Currently being treated with any ivacaftor containing regimen
研究组 & 干预措施
P-1037 in Hypertonic Saline (HS)
P-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID
干预措施: P-1037 (Drug)
P-1037 in Hypertonic Saline (HS)
P-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID
干预措施: Hypertonic Saline (Drug)
P-1037 in Saline
P-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID
干预措施: P-1037 (Drug)
P-1037 in Saline
P-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID
干预措施: Saline (Drug)
Saline
Placebo (0.17% saline) BID
干预措施: Saline (Drug)
Hypertonic Saline
Hypertonic saline (4.2% saline) BID
干预措施: Hypertonic Saline (Drug)
结局指标
主要结局
Safety (number of adverse events of P-1037 in treatment groups)
时间窗: Day 15
number of adverse events of P-1037 in treatment groups
Safety [change in forced expiratory volume in one second (FEV1) from predosing to 1 hour post dosing]
时间窗: Day 1
change in FEV1 from predosing to 1 hour post dosing with P-1037 in 4.2 % hypertonic saline and P-1037 in 0.17% saline
次要结局
- Effect of P-1037 on other forced vital capacity (FVC) measures (change in FVC from baseline (immediately before first dose) to same time of day on Day 15)(Day 15)
- Effect of P-1037 on (forced expiratory flow at 25%-75% of vital capacity (FEF25-75%)(Day 15)
- Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R)(Day 15)
- Determine whether co-administration of hypertonic saline (HS) enhances the effect of P-1037 on FEV1 (FEV1 on Day 15)(Day 15)
- Absolute change in FEV1(Day 15)