Efficacy and Safety of Omega-3 Lipid Therapy in Pediatric Patients With Parenteral Nutrition-Associated Liver Disease
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Improvement of liver dysfunction as measured by time to achieve 50 % decrease in direct bilirubin
研究概览
简要总结
This pilot study seeks to demonstrate the efficacy of an intravenous lipid preparation high in omega-3 fatty acids (Omegaven) in the treatment of cholestasis in parenteral nutrition dependent patients with short gut syndrome.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 1 Year（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patient will be dependent upon parenteral nutrition (PN)
- •Patient will have short gut syndrome (loss of >50% of small bowel)
- •Patient's guardian/caregiver provides informed consent for patient to receive therapy
- •Pediatric patient ≤ 1 year of age
- •Expected PN duration is greater than 30 days
- •Direct bilirubin >2.0 mg/dL measured on two occasions no more than one week apart
排除标准
- •Liver dysfunction secondary to cause other than PN verified by standard of care diagnostic procedures and lab work to rule out alternative causes of neonatal cholestasis.
- •Any patient in whom Omegaven therapy would be contraindicated, such as an allergy to any seafood product, egg protein, and/or previously established allergy to Omegaven
- •impaired lipid metabolism
- •severe hemorrhagic disorder
- •unstable diabetes mellitus
- •collapse and shock, stroke/embolism, recent cardiac infarction, or undefined coma status
研究组 & 干预措施
Omegaven Therapy
After baseline labs, which have been collected no earlier than seven days prior to the initiation of therapy are obtained, therapy with Omegaven will be initiated at a starting dose of 0.5 g/kg/day infused over 12 hours. If tolerated, the dose will be increased to 1 g/kg/day, the goal dose. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition.
干预措施: Omegaven Therapy (Drug)
结局指标
主要结局
Improvement of liver dysfunction as measured by time to achieve 50 % decrease in direct bilirubin
时间窗: weekly then biweekly data collection
Direct bilirubin will be collected at baseline, then weekly for 30 days, and then biweekly, thereafter, up to an expected average of 108 weeks
次要结局
- a) Maintenance of nutritional status(Labwork will be collected at baseline, then weekly for the first month. Thereafter, a lipid panel will be collected every 2 months, complete metabolic panel every 2 weeks, and essential fatty acid profile monthly, up to an expected average of 108 weeks)
- Occurrence of potential adverse side effects(biweekly labwork up to an expected average of 108 weeks)
- c) Resolution of liver dysfunction(weekly complete metabolic panel for the first month and then biweekly thereafter, up to an expected average of 108 weeks)