Inhaled Sodium Pyruvate for the Treatment of Cystic Fibrosis. A Phase I, Double Blind, Placebo Controlled, Safety Study.
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 70
- 试验地点
- 2
- 主要终点
- The primary outcome variable is the assessment of safety of inhaled sodium pyruvate in subjects with CF.Subjects will be evaluated for the presence of symptoms and safety laboratory measurements.
研究概览
简要总结
The primary objective of this study is to assess the safety of inhaled sodium pyruvate in people with Cystic Fibrosis (CF). Further, to determine whether inhaled sodium pyruvate will improve lung function, as determined by FEV1, or reduce inflammatory markers in induced sputum of people with CF.
详细描述
Cystic fibrosis (CF) is a disease that causes airway blockage and infection that damages the lung. The lungs of CF patients are frequently loaded with inflammatory cells, damaging proteins and oxidants. Oxidants are molecules that contain oxygen and are capable of disrupting cells and tissue. The CF protein is involved in the transport of the important antioxidant glutathione (GSH). Antioxidants are molecules that block oxidants and render them inactive. The absence of this protection in the airways makes them prone to damage from oxidants. Sodium pyruvate is part of the body's natural anti-oxidant defense system. Sodium pyruvate reacts directly with oxygen radicals to neutralize them and increases cellular levels of glutathione.
The purpose of this study is to investigate the safety and tolerability of 3 different single dose levels of sodium pyruvate administered via a nebulizer to persons with CF lung disease.
This will be a phase I study with 3 stages. In the first stage subjects will receive one of 3 possible doses of the active drug only once. In the second stage subjects will receive 2 doses of the active drug 12 hours apart. In the third stage subjects will receive either active drug or placebo every 12 hours for 4 weeks.
For stage 1 subjects will come to the University of Minnesota's General Clinical Research Center (GCRC) for a screening visit. They will return to the GCRC 1 to 7 days later for a baseline assessment that will be followed by study drug administration. The subject will be observed for 4 hours and safety parameters will be obtained.
For stage 2 subjects will come to the GCRC for a screening visit. They will return to the GCRC 1 to 7 days later for a baseline assessment that will be followed by study drug administration. Safety parameters will be obtained for the following 4 hours. 12 hours after the first dose was given the subject will receive a second dose and safety parameters will again be obtained over the next 4 hours.
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
- Double
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
- •Clinical diagnosis of CF using Cystic Fibrosis Foundation criteria.
- •FEV1 >40% predicted
- •Colonization with Pseudomonas aeruginosa - (>= 2 positive cultures over past 12 months)
- •>18 years of age
- •Stable respiratory status without dyspnea at rest
- •Non-smoker
- •Able to perform sputum induction
排除标准
- •Severe CF lung disease with an FEV1 of <40% predicted
- •Lung disease not CF related
- •Positive culture for Burkholderia cepacia over previous 2 years
- •Active allergic bronchopulmonary aspergillosis
- •Clinically significant cardiac disease
- •Pregnancy
- •Females of child bearing age not using contraception
- •Females lactating
- •<18 years of age
- •Systemic steroid treatment within 1 month
- •Hospitalization within 3 months due to airway disease
- •Immunotherapy
- •Changes in respiratory medication use within 1 month
- •New medications within 1 month
- •Administration of any investigational drug or device within 28 days of visit 1 or within 6 half-lives of the investigational drug (whichever is longer).
- •History of significant (>60 cc) hemoptysis within 1 year
- •Poorly controlled insulin dependent diabetes mellitus
- •Acute respiratory illness within 1 month
- •Use of tobacco products or recreational drugs
- •History of adverse reaction to sputum induction
结局指标
主要结局
The primary outcome variable is the assessment of safety of inhaled sodium pyruvate in subjects with CF.Subjects will be evaluated for the presence of symptoms and safety laboratory measurements.
次要结局
- The secondary outcome variable is the determination of improvement in lungs of CF subjects as determined by measurement of FEV1 and measurement of inflammatory markers in induced sputum.