A Randomized, Open-label Multicentre Phase 3 Trial to Assess the Safety of Tobramycin Inhalation Powder Compared to Tobramycin Solution for Inhalation in Cystic Fibrosis Subjects
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 517
- 试验地点
- 13
- 主要终点
- Number of Participants With Treatment-emergent Adverse Events
研究概览
简要总结
This study compares the safety of the tobramycin solution for inhalation with the tobramycin dry powder formulation, used with a simple inhaler
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
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of cystic fibrosis
- •Male and female patients at least 6 years of age at the time of screening.
- •Forced expiratory volume in one second (FEV1) at screening must be at least 25% and less than or equal to 75% of normal predicted values for age, sex, and height based on Knudson criteria.
- •Pseudomonas aeruginosa, a type of bacteria, must be present in a sputum/deep-throat cough swab culture (or bronchoalveolar lavage [BAL]) within 6 months prior to screening and in the sputum/ deep-throat cough swab culture at the screening visit.
- •Able to comply with all protocol requirements.
- •Clinically stable in the opinion of the investigator.
- •Use of an effective means of contraception in females of childbearing potential.
- •Provide written informed consent, Health Authority Portability and Accountability Act (HIPAA) authorization (where applicable), and assent (as appropriate) prior to the performance of any study-related procedure.
排除标准
- •History of sputum culture or deep-throat cough swab (or BAL) culture yielding Burkholderia cepacia (B cepacia), a type of bacteria, within 2 years prior to screening and/or sputum culture yielding B cepacia at screening.
- •Coughing up more than 60 cc of blood from the respiratory tract at any time within 30 days prior to study drug administration.
- •Known local or systemic hypersensitivity to aminoglycosides or inhaled antibiotics.
- •Females who are pregnant (positive pregnancy test), lactating, or are planning to become pregnant during the study.
- •History of hearing loss or chronic ringing in the ears deemed clinically significant by the investigator.
- •Use of systemic or inhaled antipseudomonal antibiotics within 28 days prior to study drug administration.
- •Use of loop diuretics within 7 days prior to study drug administration.
- •Use of any investigational treatment within 28 days prior to study drug administration.
- •Initiation of treatment with chronic macrolide therapy, dornase alpha treatment or inhaled corticosteroids within 28 days prior to study drug administration (patients may be taking these therapies at the time of enrollment, but they must have initiated treatment more than 28 days prior to study drug administration).
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
Tobramycin inhalation powder (TIP)
Participants received four 28 mg capsules of tobramycin inhalation powder (TIP) delivered with the T-326 inhaler twice daily for 28 days followed by 28 days off therapy (one cycle) for a total of three cycles.
干预措施: Tobramycin Inhalation Powder (Drug)
Tobramycin solution for inhalation (TOBI)
Participants received one 300 mg (in 5 mL) ampoule of tobramycin solution for inhalation (TOBI) delivered with a nebulizer twice daily for 28 days followed by 28 days off therapy (one cycle) for a total of three cycles.
干预措施: Tobramycin Solution for Inhalation (Drug)
结局指标
主要结局
Number of Participants With Treatment-emergent Adverse Events
时间窗: 25 weeks
An adverse event (AE) is any untoward medical occurrence, including any unfavorable and unintended sign, symptom or disease temporally associated with the use of the study medication that does not necessarily have a causal relationship with study medication. A serious AE (SAE) is an event that results in death, is life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant disability, is a congenital anomaly or defect, or is a significant medical event that may jeopardize the patient or require intervention to prevent one of the outcomes listed above.
次要结局
- Serum Tobramycin Concentrations(Weeks 1, 5, 17 and 21)
- Percentage of Participants With a Decrease From Baseline in Auditory Acuity(Baseline and Day 28 of Cycles 1, 2 and 3 (Weeks 5, 13 and 21))
- Relative Change From Baseline in Percent Predicted Forced Expiratory Volume in One Second (%FEV1)(Baseline and Day 28 of Cycles 1, 2 and 3 (Weeks 5, 13 and 21) and Final Visit (Week 25))
- Patient Satisfaction Assessed Using the Treatment Satisfaction Questionnaire for Medication(Day 28 of Cycles 1, 2 and 3 (Weeks 5, 13 and 21).)
- Change From Baseline in Pseudomonas Aeruginosa Sputum Density(Baseline and Day 28 of Cycles 1, 2 and 3 (Weeks 5, 13 and 21) and Final Visit (Week 25).)
- Change From Baseline in Tobramycin Minimum Inhibitory Concentration(Baseline and Day 28 of Cycles 1, 2 and 3 (Weeks 5, 13 and 21) and Final Visit (Week 25))
- Antipseudomonal Antibiotic Usage During the Study(25 Weeks)
- Hospitalization Due to Respiratory Events During the Study(25 Weeks)