A Prospective, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study of Nitric Oxide for Inhalation in the Acute Treatment of Sickle Cell Pain Crisis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- Mallinckrodt
- 入组人数
- 150
- 试验地点
- 11
- 主要终点
- Time to Vaso-occlusive Pain Crisis (VOC) Resolution
研究概览
简要总结
This study will examine whether nitric oxide (NO) gas can reduce the time it takes for pain to go away in patients who are in sickle cell crisis. NO is important in regulating blood vessel dilation, and consequently, blood flow. The gas is continuously produced by cells that line the blood vessels. It is also transported from the lungs by hemoglobin in red blood cells.
Patients 10 years of age or older with sickle cell disease (known SS, S-beta-thalassemia or other blood problems causing sickle cell disease) may be eligible for this study. Patients whose disease is due to hemoglobin (Hgb) SC are excluded. Candidates are screened with blood tests and a chest x-ray to look at the lungs and heart.
Participants are admitted to the hospital in a pain crisis. They are evaluated and then randomly assigned to receive one of two treatments: 1) standard treatment plus NO, or 2) standard treatment plus placebo. The placebo used in this study is nitrogen, a gas that makes up most of the air we breathe and is not known to help in sickle cell disease.
For the first 8 hours of the study, patients receive placebo or NO through a facemask. The mask may be taken off for 5 minutes every hour and for not more than 20 minutes to eat a meal. After the first 8 hours, the gas is delivered through a nasal cannula (small plastic tubing that rests under the nose) that may be taken off only while showering or using the restroom. Patients are questioned about the severity of their pain when they start the study and then every few hours while they are in the hospital. Their vital signs (temperature, breathing rate, and blood pressure) and medicines are checked. Patients will breathe the gas for a maximum of 3 days, but will stay hospitalized until the patient feels well enough to go home. Patients are followed up about 1 month after starting the study by a return visit to the hospital or by a phone call.
详细描述
The object of this study is to determine the safety and efficacy of nitric oxide for inhalation in the treatment of vaso-occlusive pain crisis (VOC) in patients with sickle cell disease. The study population will include patients with sickle cell disease (SS, S-beta-Thalassemia) presenting with vaso-occlusive pain crisis. Patients will be administered either placebo or inhaled nitric oxide to see if the experimental agent, inhaled nitric oxide, can reduce the time it takes for resolution of the vaso-occlusive crisis.
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
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Each subject must meet all of the following inclusion criteria during the screening process in order to participate in the study:
- •Patient must have a diagnosis of SCD (known SS, S-Beta-thalassemia or other hemoglobinopathies causing sickle cell disease). Patients with disease due to Hgb SC are not permitted.
- •Must present to the ED/EC or other appropriate unit in VOC.
- •Greater than or equal to 10 years old.
- •Written informed consent/assent has been obtained.
排除标准
- •Subjects meeting any of the following criteria during baseline evaluation will be excluded from entry into the study:
- •Exposure to therapeutic nitric oxide within the past 12 hours.
- •Patient has received sildenafil or other phosphodiesterase 5 inhibitors, therapeutic L-arginine, nitroprusside or nitroglycerine within the past 12 hours.
- •Patient has received previous ED/EC or other appropriate unit treatment for a vaso-occlusive crisis less than 48 hours or hospitalization less than 14 days ago (patients transferred directly from another ED or clinic may be enrolled).
- •Patient has visited the ED/EC or other appropriate unit greater than 10 times in the past year having a vaso-occlusive crisis.
- •Patients presenting with clinically diagnosed bacterial infection (e.g., osteomyelitis, pneumonia, sepsis or meningitis).
- •Patients who are currently enrolled in any other investigational drug study except for hydroxyurea studies.
- •Pregnant women (urine HCG + )/ nursing mothers.
- •Patients who have received an exchange transfusion (not simple transfusion) in the last 30 days or are on a chronic simple or exchange transfusion program.
- •Suspected splenic sequestration.
- •Acute chest syndrome or pneumonia: Abnormal new pulmonary infiltrate (alveolar infiltration and not atelectasis) and one or more pulmonary signs and/or symptoms (fever, rales, wheezing, cough, shortness of breath, retractions).
- •Previous participation in this study.
研究组 & 干预措施
Inhaled Nitric Oxide
Participants receive Inhaled nitric oxide (INO)
干预措施: Nitric Oxide (Drug)
Placebo
Participants receive Nitrogen gas
干预措施: Placebo (Drug)
结局指标
主要结局
Time to Vaso-occlusive Pain Crisis (VOC) Resolution
时间窗: within 30 days
VOC resolution was defined by all of the following conditions: * Pain relief - Visual Analog Scale (VAS) pain scores of 6 or less, (6 as worst and 0 as best) * Freedom from parenteral narcotic use, * Ability to walk unless the subject was not able to walk for any reason other than acute VOC prior to the onset of crisis, * Subject and/or family's belief that the painful crisis could be managed at home with or without oral analgesic use, and the physician concurred with that assessment.
次要结局
- Length of Hospitalization(within 40 days)
- Total Dose of Opioids Received(within 8 hours and within 40 days)
- Number of Participants With Acute Chest Syndrome/Pneumonia Requiring Blood Transfusion(within 40 days)
- Number of Participants Discharged to Home Within the First 24 Hours(within 24 hours)
- Number of Participants Readmitted to Hospital Within 30 Days After Discharge(during first 24 hours and during 30 day follow-up)