Compassionate Use of Omegaven IV Fat Emulsion in Infants With Life Threatening Parenteral Nutrition-associated Liver Disease (PNALD).
试验速览
- 阶段
- 不适用
- 状态
- 撤回
- 发起方
- 试验地点
- 1
- 主要终点
- Reduction of direct serum bilirubin level (mg/dL)
研究概览
简要总结
The purpose of the study is to provide use of Omegaven in children with life threatening Total Parenteral Nutrition (TPN) associated cholestasis when all other medical and surgical treatments/therapies have been either ineffective or not feasible in the treatment of this serious condition. This is a compassionate use protocol.
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
- 2 Months 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of life threatening PNALD
- •Dependent on Parenteral Nutrition for caloric needs
- •Expected to require PN for at least another 30 days
- •Other causes of liver disease have been excluded
- •GI/Liver service is involved in patient care
- •Standard therapies for the treatment of PNALD must have been utilized prior to initiating omegaven.
- •Inpatient at Morgan Stanley Children's Hospital of NewYork Presbyterian Hospital
- •Must be at least 2 months of age
排除标准
- •Not inpatient
- •Younger than 2 months of age
- •Expected to be weaned off of parenteral nutrition within 30 days
- •have other documented causes of liver disease
- •have signs of proven severe advanced liver disease
- •Allergy to seafood, egg protein and/or previous allergy to Omegaven
- •active coagulopathy, impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis, stoke, embolism, hemodynamic collapse or shock, recent MI, cholestasis due to any reason other than PNALD, active new infection at time of initiation of Omegaven, hemodynamic instability or if unable to tolerate the necessary laboratory monitoring
- •must not be enrolled in another clinical trial involving an investigational agent (unless approved by the designated physicians).
- •Parent or legal guardian must be willing to provide consent.
研究组 & 干预措施
Omegaven
Omegaven IV lipid emulsion administration for infants with life threatening parenteral nutrition associated liver disease
干预措施: Omegaven IV lipid emulsion (Drug)
结局指标
主要结局
Reduction of direct serum bilirubin level (mg/dL)
时间窗: Up to 6 months from the completion of the study.
For effectiveness endpoint, the allowable minimum time interval between serial direct bilirubin measurements that will determine reversal of cholestasis is 2 consecutive measurements of direct bilirubin \< or = to 2mg/dL obtained at least 2 weeks apart.
次要结局
- Essential fatty acid profile(Up to 30 days from therapy completion)
研究者
Sivan Kinberg
Assistant Professor of Pediatrics at the Columbia University Medical Center
Columbia University