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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03072667
Expanded Access Status : Approved for marketing
First Posted : March 7, 2017
Last Update Posted : December 6, 2018
St. Louis University
Information provided by (Responsible Party):
Catherine Cibulskis, MD, St. Louis University

Brief Summary:
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.

Condition or disease Intervention/treatment
Cholestasis Liver Diseases Drug: Omegaven

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Study Type : Expanded Access
Expanded Access Type : Individual Patients, Treatment IND/Protocol
Official Title: Omegaven for Compassionate Use in the Treatment of Parenteral Nutrition-Associated Liver Disease

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Liver Diseases

Intervention Details:
  • Drug: Omegaven
    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.

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 1 Year   (Child)
Sexes Eligible for Study:   All

Inclusion Criteria:

  1. Age < 1 years; inpatient use only
  2. PN dependency (unable to meet nutritional needs solely by enteral nutrition);
  3. Present PNALD, as evidenced by two consecutive direct bilirubin > 3.0 mg/dL (at interval of one week);
  4. 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.

  5. At the time the diagnosis PNALD is made, the patient is expected to continue PN at least an additional 3 weeks

Exclusion Criteria:

  1. Any other cause of chronic liver disease (i.e., hepatitis B or C, cystic fibrosis, biliary atresia, or alpha 1 anti-trypsin deficiency etc.)
  2. enrollment in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team);
  3. Lack of informed consent;
  4. Intent to transfer to another healthcare facility
  5. allergy to any seafood product, egg protein, and/or previous allergy to Omegaven
  6. active coagulopathy characterized by ongoing bleeding or by a requirement for clotting factor replacement (e.g. fresh frozen plasma or cryoprecipitate) to maintain homeostasis
  7. impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis
  8. unstable DM or hyperglycemia
  9. stroke/embolism
  10. collapse and shock
  11. undefined coma status
  12. recent MI
  13. cholestasis due to any reason other than PNALD
  14. active new infection at time of initiation of Omegaven
  15. hemodynamic instability
  16. unable to tolerate necessary laboratory monitoring
  17. severe renal insufficiency

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03072667

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United States, Missouri
SSM Health Cardinal Glennon Children's Hospital
Saint Louis, Missouri, United States, 63104
Sponsors and Collaborators
Catherine Cibulskis, MD
St. Louis University

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Responsible Party: Catherine Cibulskis, MD, Assistant Professor of Pediatrics, St. Louis University Identifier: NCT03072667    
Other Study ID Numbers: 16150
First Posted: March 7, 2017    Key Record Dates
Last Update Posted: December 6, 2018
Last Verified: December 2018
Keywords provided by Catherine Cibulskis, MD, St. Louis University:
bowel atresia
necrotizing entercolitis
parenteral nutrition associated liver disease (PNALD)
intestinal failure
parenteral nutrition associated cholestasis
fish oil
Additional relevant MeSH terms:
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Liver Diseases
Digestive System Diseases
Bile Duct Diseases
Biliary Tract Diseases