Standardizing Treatments for Pulmonary Exacerbations: A Platform for Evaluating Treatment Decisions to Improve Outcomes (STOP360) Aminoglycoside Study (AG Study)
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- 入组人数
- 730
- 试验地点
- 60
- 主要终点
- Incidence of Adverse Events (AEs) in Aminoglycoside (AG) Study Intervention Arms
研究概览
简要总结
The purpose of this study is to look at pulmonary exacerbations in people with cystic fibrosis (CF) that need to be treated with antibiotics given through a tube inserted into a vein (intravenous or IV). A pulmonary exacerbation is a worsening of respiratory symptoms in people with CF that needs medical intervention. Both doctors and CF patients are trying to understand the best way to treat pulmonary exacerbations. This study is trying to answer the following questions about treating a pulmonary exacerbation:
- Do participants have the same improvement in lung function and symptoms if they are treated with one type of antibiotic (called beta-lactams or β-lactams) versus taking two different types of antibiotics (tobramycin and β-lactams)?
- Is taking one type of antibiotic just as good as taking two types?
详细描述
Cystic Fibrosis Foundation (CFF) treatment guidelines for the management of pulmonary exacerbations (PEx) identified evidence gaps in current clinical best practices. The STOP program offers a platform for the conduct of controlled trials to develop the evidence base in order to define clinical best practices. The interventional Aminoglycoside Study (AG Study) will be a prospective, multi-center, parallel group, randomized (1:1 ratio), open-label, superiority study of intravenous aminoglycoside and β-lactams versus intravenous β-lactams only. Randomization will occur at Visit 1. The primary objective of this platform trial is to evaluate the efficacy and safety of differing treatments in CF PEx during a planned 14 day course of IV antimicrobials. Primary efficacy will be evaluated as the difference in mean Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) changes from Visit 1 to Visit 2 (Day 28 ± 2 days) between intervention arms.
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
- •All genders ≥ 6 years of age at Visit 1
- •Documentation of a CF diagnosis
- •Clinician intent to treat index CF PEx with a planned 14-day course of IV antimicrobials
- •At least one documented Pa positive culture within two years prior to Visit 1
排除标准
- •Participant is not pregnant
- •No known renal impairment or history of solid organ transplantation
- •No IV antimicrobial treatment, ICU admission, pneumothorax, or hemoptysis within 6 weeks prior to Visit 1
- •No use of investigational therapies, new CF transmembrane conductance regulator (CFTR) modulators, or treatment for Nontuberculous mycobacteria (NTM) within 4 weeks prior to Visit 1
- •No history of hypersensitivity, vestibular, or auditory toxicity with aminoglycosides
- •No more than one day of IV aminoglycosides administered for the current PEx treatment prior to Visit 1
研究组 & 干预措施
β-lactam Only (Non-AG)
Participants randomized to this arm will be prescribed a standard of care intravenous (IV) β-lactam as selected by their treating physician. Treatment must not include an IV aminoglycoside.
干预措施: Beta-lactam antibiotic (Drug)
β-lactam and Aminoglycoside (AG)
Participants randomized to this arm will be prescribed a standard of care intravenous (IV) β-lactam and aminoglycoside selected by their treating physician.
干预措施: Beta-lactam antibiotic (Drug)
β-lactam and Aminoglycoside (AG)
Participants randomized to this arm will be prescribed a standard of care intravenous (IV) β-lactam and aminoglycoside selected by their treating physician.
干预措施: Aminoglycoside (Drug)
结局指标
主要结局
Incidence of Adverse Events (AEs) in Aminoglycoside (AG) Study Intervention Arms
时间窗: Six Weeks
Difference between aminoglycoside (AG) study intervention arms (AG - Non-AG) in the proportion of participants with at least one AE from Week 0 (Day 0) to Week 6 (Day 44 ± 2 days).
Absolute Change in FEV1 % Predicted at Week 4 in Aminoglycoside (AG) Study
时间窗: Four weeks
Difference between aminoglycoside (AG) study intervention arms (AG - Non-AG) in the absolute change in FEV1 % predicted from Week 0 (Day 0) to Week 4 (Day 28 ± 2 days)
次要结局
- Absolute Change in CFRSD-CRISS Score at Week 4 in Aminoglycoside (AG) Study(Four weeks)
研究者
Chris Goss
Associate Director, UW Adult CF Center
Seattle Children's Hospital