Compassionate Use of an Intravenous Fat Emulsion Comprised of Fish Oil in the Treatment of Parenteral Nutrition Induced Liver Injury in Infants
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 293
- 试验地点
- 2
- 主要终点
- All Cause Mortality During the Study.
研究概览
简要总结
To provide a mechanism for critically ill infants with parenteral nutrition (PN) associated cholestasis to receive Omegaven for compassionate use situations for which there are no satisfactory alternative treatments.
详细描述
Inpatient Use of Omegaven:
Each family will have the severity of their child's condition explained to them and the available data regarding the benefit of the novel preparation explained using the preliminary safety and efficacy data from Boston Children's Hospital and TCH. Families will have the potential risks and benefits of the product explained. The lack of long-term follow-up information regarding the effects of Omegaven will be discussed.
Bottles containing 50mL or 100 mL of 10% Omegaven will be purchased from International Pharmacy of Hamburg, Germany. Omegaven is manufactured by Fresenius Kabi AG, Bad Homburg v.d.h, Germany. Omegaven is formulated as an emulsion from fish oils. The family (or legal guardian) will be billed for the Omegaven as part of their hospital bill from Texas Children's Hospital.
Omegaven will be dispensed per Texas Children's Hospital policy and procedures for fat emulsions.
All study materials will be stored securely until the time of administration. The bottles will be stored at room temperature below 30° C (do not freeze). Damaged or suspect drug will be returned unused to Fresenius- Kabi. Containers should be shaken before use.
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
盲法说明
Open Label
入排标准
- 年龄范围
- 14 Days 至 5 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Be greater than 14 days old and less than 5 years old
- •Conjugated bilirubin greater than 2 mg/dL.
- •Be expected to require intravenous nutrition for at least an additional 28 days
排除标准
- •Have a congenitally lethal condition (e.g. Trisomy 13).
- •Have clinically severe bleeding not able to be managed with routine measures.
- •Have evidence of a viral hepatitis or primary liver disease as the primary etiology of their cholestasis.
- •Have other health problems such that survival is extremely unlikely even if the infant's cholestasis improves.
- •Home Use of Omegaven®:
- •In order for a subject to receive the Omegaven® at home through a home health care agency, subjects will first be required to be admitted to Texas Children's Hospital for 72 hours in initiate the administration of the Omegaven®. This will allow time for observation of any unexpected side effects and for parents to be provided education on home TPN and Omegaven®.
- •If a subject has already received Omegaven® either at TCH or at another hospital, they will not be required to be admitted for the 72 hour inpatient admission prior to starting Omegaven® at home. Parent training will occur during the previous hospital admission and will continue through the TCH Pediatric Intestinal Rehabilitation Clinic.
- •Outpatient Monitoring:
- •After the initial evaluation by the TCH Pediatric Intestinal Rehabilitation Clinic physicians, subjects will return to the clinic for routine follow-up. Subjects will be asked to return to the clinic every 2 weeks for the first 2 months of treatment. Thereafter, subjects will return to the clinic on a monthly basis, or as directed by the clinic team.
研究组 & 干预措施
Fish Oil Emulsion Arm
In infants who meet the eligibility criteria for Fish Oil Emulsion arm will receive Fish Oil Emulsion after enrollment under the study.
Therapy with Fish Oil Emulsion (Omegaven) will be provided at a dose of 1 gm/kg/day (by continuous infusion) and will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition. If previously on Intralipid, it will be stopped prior to initiation of Fish Oil Emulsion.Fish oil emulsion will be provided as a continuous intravenous emulsion over 24 hours.
干预措施: Omegaven (Drug)
结局指标
主要结局
All Cause Mortality During the Study.
时间窗: From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).
To describe proportion of infants who died secondary to any cause, related or unrelated to Fish Oil Emulsion.
Growth Z-scores for Weight
时间窗: From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).
The Z-score indicated the number of standard deviations away from the mean. A weight Z-score of 0 is equal to the mean. A weight Z-score of ≤ -2 indicates an underweight status, while a weight Z-score of ≥ 2 indicates overweight or obese status.
Time to Resolution of Parenteral Nutrition Associated Cholestasis Prior to End of Study
时间窗: From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).
Time in days from the initiation of fish oil emulsions (initiation of study) until resolution of cholestasis as defined by serum conjugated bilirubin≤ 2 mg/dL prior to EOS (end of study).
次要结局
- Number of Subjects With INR ≥1.4.(From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).)
- Number of Infants Who Achieve Resolution of Parenteral Nutrition Associated Cholestasis(From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).)
- Number of Subjects With Platelet Count <100,000/µL(From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).)
- Liver or Multi-visceral Transplant(From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).)
- All-cause Mortality up to Hospital Discharge(Anytime from initiation of study to discharge from the hospital.)
- Number of Study Subjects Who Experienced a Blood Stream Infection(From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).)
研究者
Muralidhar Premkumar
Assistant Professor
Baylor College of Medicine