A Randomized, Controlled, Double-blind Clinical Trial of L-arginine as Adjuvant Therapy for Sickle Cell Disease
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 40
- 试验地点
- 1
- 主要终点
- Tricuspid regurgitant jet velocity >2.5 m/s
研究概览
简要总结
One of the main problems in sickle cell disease is the decreased bioavailability of nitric oxide and arginine. This study was designed to assess if treating sickle cell disease patients with L-arginine would improve pulmonary arterial pressure and other aspects.
详细描述
This is a phase III, randomized, double-blind, placebo-controlled clinical trial with sickle cell disease patients older than 1 year of age. The patients were randomly assigned to take 0.1 g/kg/day of either L-arginine or placebo orally. Adverse events were monitored by a safety committee. The variables were assessed while patients were in remission, as part of their routine care: weight, blood pressure, full blood cell count, creatinine and dosage of methemoglobin at baseline and at each follow-up visit. Peripheral oxygen saturation (SpO2), fetal hemoglobin, lactate dehydrogenase and tricuspid regurgitant jet velocity were measured through transthoracic Doppler echocardiogram before and after treatment.
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
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of sickle cell disease confirmed through hemoglobin electrophoresis
- •Genotypes SS, SC and Sβ thalassemia
- •Age > 1 year
排除标准
- •Liver dysfunction (alanine aminotransferase ALT > 3 times the normal levels
- •Renal dysfunction (creatinine > twice the normal levels)
- •Increase in methemoglobin levels (> 5 times the normal levels)
- •History of recent stroke (< 1 month) and priapism
- •Pregnancy
- •Allergy to L-arginine
- •Use of sildenafil, calcium channel blockers, nitroglycerin or other nitrates
研究组 & 干预措施
L-arginine
0.1g/kg/day for 6 months
干预措施: L-arginine (Drug)
L-arginine
0.1g/kg/day for 6 months
干预措施: Placebo (Drug)
结局指标
主要结局
Tricuspid regurgitant jet velocity >2.5 m/s
时间窗: 6 months
Tricuspid regurgitant jet velocity was used to assess pulmonary arterial hypertension before and after treatment with L-arginine.
次要结局
- Lactate dehydrogenase levels(6 months)