Safety and Efficacy of Glutamine in Preventing Vaso-occlusive Crisis Among Sickle Cell Disease Patients: Randomized Controlled Study
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 60
- 试验地点
- 2
- 主要终点
- Number of pain crises
研究概览
简要总结
Prospective phase IV interventional open label randomized controlled trial to assess safety and efficacy of glutamine in preventing vaso-occlusive crisis (VOC) episodes in sickle cell pediatrics and adolescents' patients
详细描述
Vaso-occlusive crisis (VOC) episodes are considered to be the cause of 95% of hospitalizations for sickle cell disease (SCD) patients. Prior studies have described pain management in SCD patients with poor outcomes in the short term and decreased quality of life in patients over the long term.
Although that L-glutamine has been recently approved by the FDA for the prevention of acute complications in sickle cell disease. However, there are many gaps in our understanding of its therapeutic implications in SCD. This study will assess the safety and efficacy of glutamine in preventing vaso-occlusive crisis (VOC) episodes in sickle cell pediatrics and adolescents' patients
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 2 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children and adolescents diagnosed with sickle cell disease by haemoglobin electrophoresis and had at least two pain crises (no upper limit) documented during the previous year; a pain crisis is defined as pain leading to treatment with a parenteral administered narcotic or ketolac in an emergency department (ED) (or outpatient treatment centre) or during hospitalization.
- •Patients receiving hydroxyurea at a fixed dose for at least 3 months before screening.
排除标准
- •Patients with sickle cell trait and other hemoglobinopathy.
研究组 & 干预措施
cases
• 30 patients will receive glutamine in a dose of 0.3 gm /kg/dose twice daily orally (up to a maximum of 15 g/dose) for 24 weeks as an add on to the SOC
干预措施: L-Glutamine, Oral Powder for Reconstitution (Drug)
control
30 patients will be assigned as a control group to receive standard of care therapy without glutamine intake.
干预措施: Standard of care (Other)
结局指标
主要结局
Number of pain crises
时间窗: 24 weeks
The number of pain crises will be counted from day 1 till end of treatment at week 24
次要结局
- Changes in transcranial doppler(24 weeks)
研究者
Fatma Soliman Elsayed Ebeid, MD
professor of pediatric hematology ,oncology and bone marrow transpalnt
Ain Shams University