Phase 2 Randomized Control Trial of Arginine Therapy for Pediatric Sickle Cell Disease Pain
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 108
- 试验地点
- 3
- 主要终点
- Total Parenteral Opioid Use in IV Morphine Equivalents
研究概览
简要总结
The aim of this study is to determine whether giving extra arginine, a simple amino acid, to patients with sickle cell disease seeking treatment for a pain crisis (vaso-occlusive painful events (VOE) will decrease pain scores, decrease the need for pain medications or decrease length of hospital stay or emergency department visit. Funding Source - FDA OOPD.
详细描述
The purpose of this study is to determine the effects of IV L-arginine hydrochloride therapy in children with sickle cell disease (SCD) and vaso-occlusive pain events (VOE). Specifically, the impact on total opioid use (mg/kg) over the duration of their emergency department (ED) visit and hospital stay will be evaluated.
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
- 3 Years 至 21 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Established diagnosis of sickle cell disease (SCD); all genotypes
- •Pain requiring medical care in an acute care setting (such as the emergency department or ED, hospital ward, day hospital, clinic) not attributable to non-sickle cell causes, that is moderate-to-severe requiring parenteral opioids
排除标准
- •Decision to discharge home from the acute care setting
- •Hemoglobin less than 5 gm/dL or immediate need for red cell transfusion anticipated within next 12 hours
- •Hepatic dysfunction of SGPT greater than 3 times the upper value
- •Renal dysfunction of creatinine greater than 1.0
- •Mental status or neurological changes
- •Acute stroke or clinical concern for stroke
- •Pregnancy
- •Allergy to arginine
- •Two (2) or more ED visits for VOE within the last 7 days prior to CURRENT ED visit
- •Hospitalization within 14 days
- •Previous randomization in this arginine RCT (patient consented and screen failed before receiving study drug or placebo remains eligible for future participation).
- •Use of inhaled nitric oxide, sildenafil or arginine within the last month
- •PICU admission from the emergency department
- •Hypotension requiring treatment with clinical intervention
- •Acidosis with Co2≤ 16
- •Newly started on HU for <3 months
- •Not an appropriate candidate in the investigator's judgment
- •Patient refusal
研究组 & 干预措施
L-Arginine
Participants will be randomized to receive an intravenous (IV) infusion of L-arginine (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital.
干预措施: L-arginine (Drug)
Loading Dose and L-Arginine
Participants will be randomized to receive an intravenous (IV) infusion of one-time loading dose of L-arginine (200 mg/kg) followed by standard dose (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital.
干预措施: L-arginine (Drug)
Loading Dose and L-Arginine
Participants will be randomized to receive an intravenous (IV) infusion of one-time loading dose of L-arginine (200 mg/kg) followed by standard dose (100 mg/kg) three times a day until time of discharge from the emergency department (ED) or hospital.
干预措施: L-arginine Loading Dose (Drug)
Placebo
Participants will be randomized to receive an intravenous (IV) infusion of placebo (normal saline 1-2 ml/kg) three times a day until time of discharge from the emergency department (ED) or hospital.
干预措施: Placebo (Other)
结局指标
主要结局
Total Parenteral Opioid Use in IV Morphine Equivalents
时间窗: Post study drug delivery to discharge from the hospital (Up to 8 days)
The total amount of parenteral opioids used by participants measured in mg/kg of IV morphine equivalents. The total is calculated after study drug delivery for participants in the emergency department (ED) and during hospital stay.
次要结局
- Time to Vaso-occlusive Pain Event (VOE) Resolution in Emergency Department(Post study drug delivery (Up to 8 hours))
- Total Number of Study Drug Doses(Duration of study (Up to 8 days))
- Rate of Acute Chest Syndrome(Duration of study (Up to 8 days))
- Rate of Return Visits to Emergency Department (ED) Within 72 Hours(Post hospital discharge (within 72 hours))
- Total Opioid Dose (ORAL + Parenteral) in mg/kg IV Morphine Equivalents(Post study drug delivery up to hospital discharge (Up to 8 days))
- Rate of Hospital Re-admissions With 30 Days(Post hospital discharge (within 30 days))
- Rate of Emergency Department (ED) Discharge(Post emergency department admission (Up to 24 hours))
- Rate of Hospital Re-admissions Within 72 Hours(Post hospital discharge (within 72 hours))
- Time to Vaso-occlusive Pain Event (VOE) Resolution in Hospital(Post study drug delivery until discharge (up to 8 days))
- Oxygen Saturation Level(At time of hospital admission and at time of Hospital discharge (Up to 8 days))
- Length of Hospital Stay(Discharge (Up to 8 days))
- Change in Vaso-occlusive Pain (VOE) Scores(Baseline, Time of discharge (Up to 8 days))
- Length of Emergency Department (ED) Stay(Until discharge or Hospital Admission (Up to 24 hours))
- Rate of Blood Transfusion(Duration of study (Up to 8 days))
- Rate of Return Visits to Emergency Department (ED) Within 30 Days(Post hospital discharge (within 30 days))
研究者
Claudia R. Morris
Professor
Emory University