Omegaven for Compassionate Use in the Treatment of Parenteral Nutrition-Associated Liver Disease
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This is a compassionate use protocol of an investigational new drug (IND). The overall purpose of the treatment is to offer alternative treatment to children who developed parenteral nutrition-associated liver disease (PNALD) and have not responded positively to currently available medical therapies. PNALD develops in newborns dependent on parenteral nutrition (PN) and are unable to tolerate adequate enteral feedings to support fluid and nutritional fluids; although PN is necessary and life sustaining, it can result in severe liver disease.
Omegaven™ will be initiated at 0.5 gm fat/kg of body weight per day (5cc/kg per day of Omegaven™) for 2 days. On day 3, the rate of Omegaven™ will be increased to 1g/kg (10cc/kg) per day and will remain at this level thereafter unless otherwise indicated.
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Ages Eligible for Study:
up to 1 Year (Child)
Sexes Eligible for Study:
Age < 1 years; inpatient use only
PN dependency (unable to meet nutritional needs solely by enteral nutrition);
Present PNALD, as evidenced by two consecutive direct bilirubin > 3.0 mg/dL (at interval of one week);
Failed standard therapies to prevent the progression of liver disease:
Avoiding overfeeding, reduction/removal of copper and manganese from PN, advancement of enteral feeding and the use of Ursodiol, if possible.
At the time the diagnosis PNALD is made, the patient is expected to continue PN at least an additional 3 weeks
Any other cause of chronic liver disease (i.e., hepatitis B or C, cystic fibrosis, biliary atresia, or alpha 1 anti-trypsin deficiency etc.)
enrollment in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team);
Lack of informed consent;
Intent to transfer to another healthcare facility
allergy to any seafood product, egg protein, and/or previous allergy to Omegaven
active coagulopathy characterized by ongoing bleeding or by a requirement for clotting factor replacement (e.g. fresh frozen plasma or cryoprecipitate) to maintain homeostasis
impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis
unstable DM or hyperglycemia
collapse and shock
undefined coma status
cholestasis due to any reason other than PNALD
active new infection at time of initiation of Omegaven
unable to tolerate necessary laboratory monitoring