A Phase III Open-Label Extension Study to Assess the Safety and Efficacy of Tobramycin Inhalation Powder After Manufacturing Process Modifications (TIPnew) in Cystic Fibrosis (CF) Patients Who Successfully Completed Participation in Study CTBM100C2303E1
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 49
- 试验地点
- 1
- 主要终点
- Number of Participants With Adverse Events (AEs)
研究概览
简要总结
This was an open-label, single arm (uncontrolled) study in participants suffering from cystic fibrosis, who have completed their study participation in CTBM100C2303 and extension study one CTBM100C2303E1 (all visits), who were proven infected with Pseudomonas aeruginosa at enrollment into CTBM100C2303.
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
- 6 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completed all visits in study CTBM100C2303 and CTBM100C2303E1, and visit 11 of study CTBM100C2303E1 took place not more than 5 days before enrollment into this study.
- •Confirmed diagnosis of cystic fibrosis participants with P. aeruginosa infection.
- •Forced Expiratory Volume in one second (FEV1) at screening (at start of study CTBM100C2303) must be between 25% and 80% of normal predicted values.
排除标准
- •Any use of inhaled anti-pseudomonal antibiotics between the termination of the trial CTMB100C2303E1 and the enrollment into this study.
- •Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics.
研究组 & 干预措施
Tobramycin Inhalation Powder (TIPnew)
Participants received four capsules of 28 mg TIPnew (112 mg), inhaled twice a day (b.i.d.) in the morning and the evening given in a cycle of 28 days on treatment followed by 28 days off treatment for three consecutive cycles.
干预措施: Tobramycin inhalation powder (Drug)
结局指标
主要结局
Number of Participants With Adverse Events (AEs)
时间窗: From time of first administration of study drug until study completion (up to 169 days)
An AE was defined as any unfavorable and unintended sign, symptom, or disease temporally associated with the use of study drug, whether or not related to study drug.
Percentage of Participants With Frequency Decrease From Baseline in the Post-baseline Audiology Tests
时间窗: Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57)
Auditory acuity of participants was measured using a standard dual-channel audiometer at frequencies from 250 to 8000 Hertz, and an audiogram (pure-tone air conduction) and tympanogram were performed by an audiologist. The categories reported includes \>= 10dB decrease in 3 consecutive frequencies in either ear, \>= 15dB decrease in 2 consecutive frequencies in either ear, and \>= 20dB decrease in at least one frequency in either ear
Number of Participants With Serious Adverse Events (SAEs)
时间窗: From time of consent to 4 weeks after study completion (up to 199 days)
A SAE was defined as an event which was fatal or life threatening, required or prolonged hospitalization, was significantly or permanently disabling or incapacitating, constituted a congenital anomaly or a birth defect, or encompassed any other clinically significant event that could jeopardize the participant or require medical or surgical intervention to prevent one of the aforementioned outcomes.
Percentage of Participants With a Decrease of ≥20% in Forced Expiratory Value in One Second (FEV1) Percent (%) Predicted From Pre-dose to 30-minute Post-dose
时间窗: Pre-dose and post-dose of Day 1 and Day 29 of every Cycle (5, 6, 7)
Airway Reactivity \>= 20% relative decrease in FEV1% predicted from pre-dose to 30 minutes post-dose. Relative Change = 100 \* (30 minutes Post-dose - Pre-dose)/Pre-dose assessed by the number and percentage of participants with a decrease of ≥ 20% in FEV1 % predicted from pre-dose to 30 minutes post-dose.
次要结局
- Number of Participants Who Used New Antipseudomonal Antibiotic During Treatment Period(Baseline, Cycles 5, 6, 7 (Days 1, 29))
- Percentage of Participants With Hospitalization Due to Respiratory Serious Adverse Events (SAEs)(From time of consent to 4 weeks after study completion (up to 199 days))
- Number of Days of Hospitalization Due to Respiratory Serious Adverse Events (SAEs)(From time of consent to 4 weeks after study completion (up to 199 days))
- Relative Change From Baseline of Forced Vital Capacity (FVC) Percent Predicted to Each Post-baseline Visit(Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57))
- Change From Baseline in Pseudomonas Aeruginosa Sputum Density to Each Post-baseline Visit(Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57))
- Change From Baseline in Tobramycin Minimum Inhibitory Concentration (MIC) Values for Pseudomonas Aeruginosa to Each Post-baseline Visit(Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57))
- Relative Change From Baseline of Forced Expiratory Flow Rate Over 25 and 75 Percent (FEF25-75%) Predicted to Each Post-baseline Visit(Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57))
- Relative Change From Baseline of Forced Expiratory Volume in One Second (FEV1) Percent Predicted to Each Post-baseline Visit(Baseline, Cycles 5, 6, 7 (Days 1, 29) and Follow-up (Week 57/Day 57))