Nitric Oxide Therapy for Acute Chest Syndrome in Sickle Cell Disease Children: Randomized, Double Blind Placebo-controlled Concept-proof Trial
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 21
- 试验地点
- 1
- 主要终点
- We will observe the patient's transfusional needs under nitric oxide (MONOXYDE AZOTE) versus placebo inhaled therapy to evaluate inhaled MOXYDE AZOTE efficacy on improving oxygenation (transcutaneous O2 superior to 92% )
研究概览
简要总结
Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis.
详细描述
Acute chest syndrome is a severe sickle cell disease complication in children requiring blood transfusion therapy to prevent acute respiratory failure and death. Nitric oxide is a potent vasodilator that could reverse pulmonary vascular occlusion and restore normal oxygenation. The randomized trial will test that hypothesis in a prospective randomized double-blind placebo controlled study. 50 children in two years will be included: 25 in each arm.
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 (Participant, Investigator)
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •child between 1 and 18 years old
- •Sickle cell anemia or equivalent (sickle beta 0 thalassemia)whoe weight is between 10kg and 65kg
- •presenting acute chest syndrome as defined a new radiological infiltrate with tachypnea, respiratory discomfort, cough, chest wall pain and fever more than 38.5°C
- •hypoxaemia with transcutaneous oxygen saturation equal or less than 92%
- •informed consent signed by parents and approved by the child able to express his consent
- •insured by the National social security system or by the universal medical insurance
- •previous medical physical examination
排除标准
- •respiratory distress with hypoxaemia with transcutaneous oxygen saturation equal or less than 92% under more than 5l/min of oxygen or 40% oxygen inhaled, hypercapnia signs 'sweating, altered consciousness, paCO2 more than 60mmHg) with need of emergency exchange transfusion and/or tracheal intubation with mechanical ventilation
- •Isolated acute asthmatic crisis
- •stroke or priapism with emergency acute transfusion needed
- •acute anemia with hemoglobin drop of more than 20% as compared to steady state hemoglobin
- •chronic long term transfusion therapy
- •nitric oxyde hypersensitivity
- •patients with right-left extra-pulmonary cardiac shunt
- •patient previously included in the protocol
- •patient participating in another interventional protocol
- •pregnancy or breast feeding
研究组 & 干预措施
1: INOMAX
Nitric oxide by inhalation INOMAX:
active arm treated with nitric oxide
干预措施: Nitric oxide by inhalation INOMAX (Drug)
2: Placebo
placebo arm treated with placebo at the same conditions
干预措施: Placebo (Drug)
结局指标
主要结局
We will observe the patient's transfusional needs under nitric oxide (MONOXYDE AZOTE) versus placebo inhaled therapy to evaluate inhaled MOXYDE AZOTE efficacy on improving oxygenation (transcutaneous O2 superior to 92% )
时间窗: Oxygenation improvement (transcutaneous O2 superior to 92%) after Gas inhalation will be evaluate 2hours after inclusion and therafter every 2 hours until 12 hours therapy and then every six hours for 3days and then once a day till hospital discharge
次要结局
- Number of blood transfusions and total transfused blood volume(7 to 10 days)
- Quantity of Pain-killer drugs required and particularly OPIOIDS(7 to 10 days)
- Duration of OXYGENOTHERAPY(7 to 10 days)
- Duration of Nitric oxide therapy(after 7 to 10 days)