Omegaven for Compassionate Use in the Treatment of Parenteral Nutrition-Associated Liver Disease

APPROVED_FOR_MARKETING

Conditions studied: Cholestasis, Liver Diseases

In brief

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.

Key facts

Study ID

NCT03072667

Run by

Catherine Cibulskis, MD

Last updated by the study team

2018-12-06

Who can join

Age: any, up to 1. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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
• stroke/embolism
• collapse and shock
• undefined coma status
• recent MI
• cholestasis due to any reason other than PNALD
• active new infection at time of initiation of Omegaven
• hemodynamic instability
• unable to tolerate necessary laboratory monitoring
• severe renal insufficiency

Where it is running

• SSM Health Cardinal Glennon Children's Hospital — St Louis, Missouri, United States

Full record on ClinicalTrials.gov

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