Randomized Controlled Trial of Prednisone in Cystic Fibrosis Pulmonary Exacerbations
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 76
- 试验地点
- 12
- 主要终点
- Lung function recovery
研究概览
简要总结
This will be a 5 year randomized, double blind, placebo controlled trial of 7 days of oral prednisone in cystic fibrosis (CF) patients receiving intravenous (IV) antibiotic treatment for a pulmonary exacerbation at the Hospital for Sick Children and other study sub-sites across Canada. The intervention will be oral prednisone 2 mg/kg/day (max 60 mg) divided twice daily for 7 days as an adjunctive therapy for pulmonary exacerbations in CF patients who have not recovered their baseline forced expiratory volume in 1 second (FEV1) after 7 days of IV antibiotic treatment. The primary outcome will be the proportion of subjects who achieve >90% of their baseline FEV1 % predicted at day 14 of IV antibiotic treatment for a pulmonary exacerbation in each treatment arm.
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)
盲法说明
placebo
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of CF by newborn screening or at least one clinical feature of CF, AND either (a) or (b) as follows:
- •A documented sweat chloride ≥ 60 mEq/L by quantitative pilocarpine iontophoresis
- •A genotype with two identifiable CF-causing mutations
- •Age > 6 years old.
- •Acute pulmonary exacerbation treated with IV antibiotics as previously defined 10% relative drop in FEV1 from baseline at the time of exacerbation
- •Informed consent by patient or parent/legal guardian
- •Ability to reproducibly perform pulmonary function testing
- •Ability to comply with medication use including the ability to take capsules, study visits and study procedures as judged by the site investigator
排除标准
- •A respiratory tract culture positive for Burkholderia cenocepacia in the 12 months prior to enrollment
- •A respiratory tract culture positive for Mycobacterium abscessus in the 12 months prior to enrollment
- •Treatment with IV or oral corticosteroids within 2 weeks of enrollment or from Day 0-Day 7 of the pulmonary exacerbation
- •Active allergic bronchopulmonary aspergillosis (ABPA) at the time of enrollment as determined by treating physician
- •Asthma related exacerbation at enrollment as defined by the treating physician based on clinically compatible symptoms (eg. wheeze)
- •History of avascular necrosis or pathologic bone fracture
- •Uncontrolled hypertension with end organ damage
- •Active gastrointestinal bleeding
- •Status post lung or other organ transplantation
- •Lactose intolerance (contained in placebo)
- •On Lumacaftor-Ivacaftor (Orkambi) at the time of exacerbation
- •Investigational drug use within 30 days prior to enrollment visit
- •Physical findings that would compromise the safety of the subject or the quality of the study data as determined by site investigator
研究组 & 干预措施
Placebo
Placebo
干预措施: Placebos (Drug)
Treatment
Prednisone
干预措施: Prednisone (Drug)
结局指标
主要结局
Lung function recovery
时间窗: At 14 days of antibiotic therapy
The proportion of subjects who achieve \>90% of their baseline FEV1 % predicted at day 14 of IV antibiotic treatment for a PEx in each treatment arm.
次要结局
- lung function recovery at follow up visit(1 month follow up)
- change in pulmonary function testing(at day 7, 14 and 1 month follow up)
- quality of life as measured by CFQ-R questionnaire(at day 7, 14 and 1 month follow up)
- quality of life as measured by CF Respiratory Symptom Diary(at day 7, 14 and 1 month follow up)
- change in sputum inflammatory markers(at day 7, 14 and 1 month follow up)
- time to subsequent pulmonary exacerbation(1 year follow up time)
- change in serum inflammatory markers(at day 7, 14 and 1 month follow up)
- number of adverse events(At day day 14 of antibiotic therapy and 1 month follow up)
- length of hospitalization(Through study completion, up to 100 weeks)
- Duration of antibiotic treatment(Through study completion, up to 100 weeks)
研究者
Valerie Waters
Staff physician
The Hospital for Sick Children