A Randomized, Double-blind, Placebo-controlled, Parallel Group Trial Evaluating Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of BI 1291583 One Tablet Once Daily Over 12 Weeks Versus Placebo in Adult Patients With Cystic Fibrosis Bronchiectasis (Clairafly™)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 22
- 试验地点
- 36
- 主要终点
- Occurrence of Any Treatment Emergent Adverse Events
研究概览
简要总结
This study is open to adults aged 18 years and older with cystic fibrosis bronchiectasis.
The purpose of this study is to find out whether a medicine called BI 1291583 is tolerated by people with cystic fibrosis bronchiectasis. Participants are put randomly into 2 groups. One group takes BI 1291583 tablets and the other group takes placebo tablets. Placebo tablets look like BI 1291583 tablets but do not contain any medicine. Participants in both groups take 1 tablet once a day for 12 weeks. Participants have twice the chance of being placed in the BI 1291583 group than in the placebo group.
Participants are in the study for about 6 months. During this time, they visit the study site 7 times. At the visits, the doctors check the health of the participants and note any health problems that could have been caused by BI 1291583.
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
- •Age of patients when signing the informed consent ≥18 years
- •Historical clinical diagnosis of Cystic fibrosis (CF) (symptoms of CF and sweat chloride ≥ 60 mmol/L and/or 2 CF-causing Cystic fibrosis transmembrane conductance regulator (CFTR) mutations)
- •Investigator-confirmed diagnosis of Bronchiectasis (BE) by Computed tomography (CT) scan and clinical history consistent with BE (e.g., cough, chronic sputum production, recurrent respiratory infections). Subjects whose past chest CT records are not available will undergo a chest CT scan during Screening. Historical scans must not be older than 5 years
- •History of pulmonary exacerbations requiring antibiotic treatment. In the 12 months before Visit 1, patients must have had either:
- •at least 2 exacerbations, or
- •at least 1 exacerbation and an St. George's Respiratory Questionnaire (SGRQ) Symptoms score of >40 at screening visit 1 For patients on stable oral or inhaled antibiotics as chronic treatment for BE, at least one exacerbation must have occurred while on stable antibiotics.
- •Patients must be able to provide spontaneous or induced sputum samples. Further inclusion criteria apply.
排除标准
- •Moderate or severe liver disease (defined by Child-Pugh score B or C hepatic impairment) or Aspartate aminotransferase (AST) and/or Alanine aminotransferase (ALT) > 3.0x Upper limit of normal (ULN) at Visit 1
- •Estimated glomerular filtration rate (eGFR) according to Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula < 30 mL/min at Visit 1
- •Absolute blood neutrophil count < 1,000/mm^3 (equivalent to < 1000 cells/μL or < 10^9 cells/L) at Visit 1
- •Any findings in the medical examination (including blood pressure (BP), pulse rate (PR), or electrocardiogram (ECG)) and/or laboratory value assessed at Visit 1 or during screening period that in the opinion of the investigator may put the patient at risk by participating in the trial
- •Positive serological tests for hepatitis B, hepatitis C (also confirmed with Hepatitis C virus ribonucleic acid (HCV RNA)), or human immunodeficiency virus (HIV) infection, or known infection status.
- •Further exclusion criteria apply.
研究组 & 干预措施
Placebo
干预措施: Placebo to BI 1291583 (Drug)
5 mg BI 1291583
干预措施: BI 1291583 (Drug)
结局指标
主要结局
Occurrence of Any Treatment Emergent Adverse Events
时间窗: From first dose of trial drug administration until last dose of trial drug administration plus 28 days of residual effect period, up to 16 weeks.
Occurrence of any treatment emergent adverse events is expressed as percentages of participants with treatment emergent adverse events. Percentages are rounded to one decimal place.
次要结局
- Relative Change From Baseline in NE Activity, in Sputum, at Week 8 After First Drug Administration(Before the first drug administration (baseline: mean value of screening and Week 0 prior to the first treatment intake) and Week 8 after first study drug administration.)
- Area Under the Curve of BI 1291583 in Plasma Over a Uniform Dosing Interval Tau=6h After the First Dose (AUC0-6)(Before drug administration and 1 hour (h), 3.5h, 6h after administration of the first dose of BI 1291583 at Day 1.)
- Area Under the Curve of BI 1291583 in Plasma Over a Uniform Dosing Interval Tau=6h at Steady State (AUC0-6,ss)(Before drug administration at Day 85 and 1 hour (h), 3.5h, 6h after administration of BI 1291583 at Day 85.)
- Maximum Measured Concentration of BI 1291583 in Plasma After the First Dose(Before drug administration and 1 hour (h), 3.5h, 6h, and 8h after administration of the first dose of BI 1291583 at Day 1.)
- Maximum Measured Concentration of BI 1291583 in Plasma at Steady State(Before drug administration at Day 85 and 1 hour (h), 3.5h, 6h after administration of BI 1291583 at Day 85.)