Compassionate Use of Omegaven in the Treatment of Parenteral Nutrition Induced Hepatic Injury
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 15
- 试验地点
- 1
- 主要终点
- Bilirubin in mg/dL
研究概览
简要总结
This study is being done to determine if an investigational study drug called Omegaven can help to improve liver disease thought to be caused by Total Parenteral Nutrition (TPN). TPN is intravenous feeding (IV feeding tube) that provides patients the nutrients when they are unable to drink fluid or eat food by mouth.
详细描述
PAtients with direct bilirubin >2gm/dL and short bowel syndrome are eligible for enrollment. Omegaven at 1gm/kg/day will be administered via central venous line instead of standard lipid preparations.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 2 Months 至 10 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients must be 2 months -10 years of age
- •Patients who are PN dependent (unable to meet nutritional needs solely by enteral nutrition) and are expected to require PN for at least another 30 days
- •Patients considered eligible for study participation must have parenteral nutrition associated liver disease (PNALD) as defined by a direct bilirubin of >2.0mg/dL. Other causes of liver disease should be excluded. A liver biopsy is not necessary for treatment
- •2 consecutive direct bilirubin test results >2.0mg/dl
- •The patient must have failed standard therapies to prevent the progression of liver disease such as surgical treatment, cyclic TPN, avoiding overfeeding, reduction/removal of copper and manganese from TPN, advancement of enteral feeding, and use of ursodiol (Actigall)
- •Subjects who are currently under treatment for PNALD with Compassionate Use Omegaven and have a direct Bilirubin of< 2, but who remain TPN dependent and require continued therapy with Omegaven
排除标准
- •Other known causes of chronic liver disease (hepatitis C, cystic fibrosis, biliary atresia and alpha 1 anti-trypsin deficiency)
- •Enrollment in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)
- •The parent, guardian, or child is unwilling to provide consent or assent
- •Allergy to any fish product, egg protein, and/or previous allergy to Omegaven
- •Active coagulopathies characterized by on-going bleeding or by a requirement for clotting factor replacement such as fresh frozen plasma or cryoprecipitate to maintain homeostasis
- •Impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis
- •Unstable diabetes mellitus
- •Stroke/embolism
- •Collapse and shock
- •Undefined coma status
- •Active infection at time of initiation of Omegaven® up until such time as child is afebrile with stable vital signs and one negative 48 hour culture
研究组 & 干预措施
Compassionate use
This lipid preparation is only being used for compassionate use.
干预措施: Omegaven (Drug)
结局指标
主要结局
Bilirubin in mg/dL
时间窗: 12 weeks
PN associated liver disease can be reversed or its progression halted by using a parenteral fat emulsion prepared from fish oil as measured by normalization of serum levels of hepatic enzymes and bilirubin 1. after reaching bilirubin levels \>2mg/dl, subjects receiving Omegaven will reach a bilirubin level \< 2mg/dL faster that patients receiving conventional fat emulsions.
次要结局
- Number of participants with adverse events(12 weeks)
研究者
Nandini Channabasappa, MD
Director, Intestinal Rehabilitation Program
Children's Medical Center Dallas