Safety and Tolerability Study of Single Dose Escalations of TR02 (SLIT™ Amikacin) by Inhalation in Cystic Fibrosis Study Patients With Chronic Infections of Pseudomonas Aeruginosa
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 18
- 主要终点
- Number of Participants who Experience a Treatment Emergent Adverse Event (TEAE)
研究概览
简要总结
The primary purpose of this study is to evaluate the safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 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
- •Study participants must produce sputum that is positive for Pseudomonas aeruginosa.
- •Confirmed diagnosis of CF (positive sweat chloride >60 milliequivalents (mEq)/liter (by pilocarpine iontophoresis) and/or a genotype with two identifiable mutations consistent with CF accompanied by one or more clinical features with the CF phenotype.
- •Forced expiratory volume (FEV1) ≥40% predicted at Screening as calculated by the Knudsen reference equations.
- •Clinically stable with no evidence of current pulmonary exacerbation.
排除标准
- •History of lung transplantation.
- •Use of intravenous antibiotics or oral quinolones within 14 days of Screening.
- •Use of low dose oral antibiotics (e.g. tetracycline, sulfa) for acne or other conditions within 30 days of Screening.
- •Use of systemic corticosteroids (≥20 milligrams [mg] of prednisone per day) within 30 days of Screening.
- •Initiation of TOBI® (tobramycin), high dose ibuprofen, recombinant human DNase (rhDNase), or macrolide antibiotics within 60 days of Screening.
- •History of sputum or throat swab culture yielding Burkholderia cepacia complex within 2 years of Screening or growth of Burkholderia cepacia complex from the sputum or throat swab culture obtained at Screening.
- •History of biliary cirrhosis, portal hypertension, or splenomegaly or splenomegaly on physical exam at Screening or enrollment.
- •History of daily continuous oxygen supplementation or requirement for more than 2 liter per minute (L/min) at night.
- •Note: Other inclusion/exclusion criteria may apply.
研究组 & 干预措施
Cohort 1: Amikacin Dose 1 + Placebo
Participants will receive a single dose of SLIT™ amikacin at Dose 1 or matching placebo by inhalation on Day 0.
干预措施: Placebo (Drug)
Cohort 1: Amikacin Dose 1 + Placebo
Participants will receive a single dose of SLIT™ amikacin at Dose 1 or matching placebo by inhalation on Day 0.
干预措施: SLIT™ Amikacin (Drug)
Cohort 2: Amikacin Dose 2 + Placebo
Participants will receive a single dose of SLIT™ amikacin at Dose 2 or matching placebo by inhalation on Day 0 upon initiation of Cohort 2.
干预措施: SLIT™ Amikacin (Drug)
Cohort 2: Amikacin Dose 2 + Placebo
Participants will receive a single dose of SLIT™ amikacin at Dose 2 or matching placebo by inhalation on Day 0 upon initiation of Cohort 2.
干预措施: Placebo (Drug)
Cohort 3: Amikacin Dose 3 + Placebo
Participants will receive a single dose of SLIT™ amikacin at Dose 3 or matching placebo by inhalation on Day 0 upon initiation of Cohort 3.
干预措施: SLIT™ Amikacin (Drug)
Cohort 3: Amikacin Dose 3 + Placebo
Participants will receive a single dose of SLIT™ amikacin at Dose 3 or matching placebo by inhalation on Day 0 upon initiation of Cohort 3.
干预措施: Placebo (Drug)
结局指标
主要结局
Number of Participants who Experience a Treatment Emergent Adverse Event (TEAE)
时间窗: Up to Day 28
Safety and tolerability of three active doses of nebulized amikacin in a SLIT™ formulation.
次要结局
- AUC of SLIT™ Amikacin in Sputum(Pre-dose and at multiple time points post-dose on Days 1, 2, 3, 8, 14, and 28)
- Change From Baseline in Sputum Density of Pseudomonas Aeruginosa(Baseline up to Day 28)
- Area Under the Concentration-time Curve (AUC) of SLIT™ Amikacin in Serum(Pre-dose and at multiple time points post-dose up to Day 3)
- Percent Dose of SLIT™ Amikacin in Urine(At multiple time points post-dose up to Day 3)