Safety and Tolerability of Inhaled Nitric Oxide in Patients With Cystic Fibrosis
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 发起方
- Mallinckrodt
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Change in Oxygen Saturation
研究概览
简要总结
The primary objective of the trial is to assess the safety and tolerability of inhaled nitric oxide (NO) when administered by nasal cannula over a 44 hour period to clinically stable Cystic Fibrosis (CF) subjects. Toxicity is to be defined as a drop in oxygen saturations, a decline in forced expiratory volume in one second (FEV1), or an increase in methemoglobin.
详细描述
Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A cycle of chronic, persistent infections with CF-related pathogens and an excessive inflammatory response progressively damages the airways and lung parenchyma, resulting in widespread bronchiectasis and ultimately, respiratory failure. Despite tremendous advances in understanding the CF gene and the CFTR protein, it is not known exactly how mutations in the gene and defects in CFTR lead to persistent airway infection and inflammation.
Inhaled nitric oxide (NO) has potential to be an effective treatment in CF lung disease. Inhaled NO has been studied in other airways diseases characterized by infection and /or inflammation such as COPD and idiopathic pulmonary fibrosis.
NO has been shown to activate CFTR and alternative chloride channels, thereby increasing chloride current in epithelial cells. Therefore, NO treatment may be beneficial in individuals with CF.
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
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Confirmed diagnosis of CF
- •12 years of age and older
- •FEV1 greater than 40% of predicted
- •Resting awake oxygen saturation of at least 88%
- •Stable pulmonary disease as defined by both clinical impression and having had no recent hospitalizations or changes in antibiotic regimen within 1 month prior to enrollment
- •Signed informed consent form
排除标准
- •Pulmonary exacerbation resulting in antibiotic treatment (except prophylactic antibiotics) within 1 month of enrollment
- •Isolation of B. cepacia from a respiratory tract culture within 6 months
- •Severe nasal obstruction at the time of screening
- •Receipt of any aerosolized experimental or investigational drugs within 1 month of enrollment
- •Pregnancy (a negative pregnancy test must be documented prior to enrollment if applicable)
- •Patients who have received treatment with nitric oxide for inhalation within 24 hours prior to study initiation or other investigational medications within 24 hours.
研究组 & 干预措施
Low Dose Cohort
Subjects in the low dose cohort receive 20 part per million (ppm) of nitric oxide via nasal cannula over a 44 hour period.
干预措施: Nitric Oxide for Inhalation (Drug)
High-Dose Cohort
Subjects in the high dose cohort receive 40 ppm of nitric oxide via nasal cannula over a 44 hour period.
干预措施: Nitric Oxide for Inhalation (Drug)
Nitrogen
100% Nitrogen (placebo) will be administer at 20 ppm or 40 ppm via nasal cannula over a 44 hour period.
干预措施: Nitrogen (Drug)
结局指标
主要结局
Change in Oxygen Saturation
时间窗: Baseline and 48 hours
Safety and tolerability of drug assessed by decreased oxygen saturation was measured through pulse oximeter, which measure the amount of oxygen in the blood. Normal range percentage is 95 - 100%
Change in Forced Expiratory Volume in 1 Second (FEV1)
时间窗: Baseline and 48 hours
Decrease in forced expiratory volume in 1 second was measured through spirometer. Spirometer measures the volume of air inspired and expired by the lungs.
Safety and Tolerability of Drug, Assessed by Change in Methemoglobin Levels
时间窗: Baseline and 48 hours
Methemoglobin level assessments were measured through blood draws - hematology. This test measures the amount of methemoglobin (a type of hemoglobin that is unable to transport oxygen to tissues) in blood. Normal methemoglobin percentage range 1% - 2%.
次要结局
未报告次要终点