A Phase 3 Multi-centre Randomised Placebo-controlled Study of Azithromycin in the Primary Prevention of Radiologically-defined Bronchiectasis in Infants With Cystic Fibrosis.
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 130
- 试验地点
- 9
- 主要终点
- Proportion of children with radiologically-defined bronchiectasis
研究概览
简要总结
The general aim of this project is to conduct a randomized, double-blind, placebo-controlled clinical trial of azithromycin to determine whether treatment from infancy is safe and will prevent the onset of bronchiectasis. One hundred and thirty infants will be recruited from CF clinics in Australia and New Zealand and treated from 3 months to three years of age. The primary outcome will be the proportion with radiologically-defined bronchiectasis at 3 years of age. Safety and mechanistic evaluations will also be undertaken.
详细描述
SYNOPSIS OF PROTOCOL
Title Multi-centre randomized placebo-controlled study of azithromycin in the primary prevention of radiologically-defined bronchiectasis in infants with Cystic Fibrosis
Clinical Phase Phase 3
Protocol Number: AZI001
TGA Reference Number:
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 6 Weeks 至 6 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children of either sex with a diagnosis of CF following detection via New Born Screening (NBS) for cystic fibrosis
- •Participants who, in the opinion of the Investigator, are able to comply with the protocol for its duration
- •Written informed consent signed and dated by parent/legal guardian according to local regulations
排除标准
- •Born <30 weeks gestation
- •Prolonged mechanical ventilation in the first 3 months of life
- •Participation in another randomized controlled trial within the 3 months preceding inclusion in this study
- •A significant medical disease or condition other than CF that is likely to interfere with the child's ability to complete the entire protocol
- •Previous major surgery except for meconium ileus
- •Macrolide hypersensitivity
研究组 & 干预措施
azithromycin liquid preparation
azithromycin will be given at a dose of 10mg/kg given three times per week from three months of age to three years of age
干预措施: Azithromycin (Drug)
inert liquid preparation
inert liquid preparation will be given three times per week from three months of age to three years of age
干预措施: Placebo control (Drug)
结局指标
主要结局
Proportion of children with radiologically-defined bronchiectasis
时间窗: at three years of age
bronchiectasis will be determined from a low dose volumetric chest computed tomography scan performed at 3 years of age
The proportion of lung tissue affected by disease
时间窗: at three years of age
Percentage of diseased lung will be determined from a low dose volumetric chest computed tomography scan performed at 3 years of age
次要结局
- body mass index(at three years of age)
- proportion of participants experiencing a pulmonary exacerbation(over the first three years of life)
- time to first pulmonary exacerbation(over the first three years of life)
- Volume of trapped gas at age 3 years(at 3 years)
- extent and severity of bronchiectasis(at three years of age)
- Proportion of participants growing Pseudomonas aeruginosa in bronchoalveolar lavage(over the first three years of life)
- age of acquisition of Pseudomonas aeruginosa(over the first three years of life)
- CF-related quality of life(at three years of age)
- Emergence of macrolide-resistant Staphylococcus aureus, small colony variant Staphylococcal aureus and non-tuberculous mycobacterium(over the first three years of life)