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

NCT03072667 | Unknown

• 1 other identifier: 16150
• Study Type: expanded_access
• Target: N/A
• Locations: 1 country
• Sites: 1

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.

Conditions

Cholestasis; Liver Diseases

Keywords

prematurity; bowel atresia; necrotizing entercolitis; parenteral nutrition associated liver disease (PNALD); intestinal failure; parenteral nutrition associated cholestasis; fish oil

Interventions

Omegaven DRUG

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.

Eligibility Criteria

• Age: Up to 1 Year
• Sex: all
• Age Groups: Child (0-17)

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

Sponsors & Collaborators

• Catherine Cibulskis, MD: lead
• St. Louis University: collaborator

Study Sites (1)

SSM Health Cardinal Glennon Children's Hospital

St Louis, Missouri, 63104, United States

Location

Related Publications (8)

• Steinbach M, Clark RH, Kelleher AS, Flores C, White R, Chace DH, Spitzer AR; Pediatrix Amino-Acid Study Group. Demographic and nutritional factors associated with prolonged cholestatic jaundice in the premature infant. J Perinatol. 2008 Feb;28(2):129-35. doi: 10.1038/sj.jp.7211889. Epub 2007 Dec 6.

  PMID: 18059467 BACKGROUND

• Alwayn IP, Gura K, Nose V, Zausche B, Javid P, Garza J, Verbesey J, Voss S, Ollero M, Andersson C, Bistrian B, Folkman J, Puder M. Omega-3 fatty acid supplementation prevents hepatic steatosis in a murine model of nonalcoholic fatty liver disease. Pediatr Res. 2005 Mar;57(3):445-52. doi: 10.1203/01.PDR.0000153672.43030.75. Epub 2005 Jan 19.

  PMID: 15659701 BACKGROUND

• Gura KM, Parsons SK, Bechard LJ, Henderson T, Dorsey M, Phipatanakul W, Duggan C, Puder M, Lenders C. Use of a fish oil-based lipid emulsion to treat essential fatty acid deficiency in a soy allergic patient receiving parenteral nutrition. Clin Nutr. 2005 Oct;24(5):839-47. doi: 10.1016/j.clnu.2005.05.020.

  PMID: 16029913 BACKGROUND

• Gura KM, Duggan CP, Collier SB, Jennings RW, Folkman J, Bistrian BR, Puder M. Reversal of parenteral nutrition-associated liver disease in two infants with short bowel syndrome using parenteral fish oil: implications for future management. Pediatrics. 2006 Jul;118(1):e197-201. doi: 10.1542/peds.2005-2662.

  PMID: 16818533 BACKGROUND

• Gura KM, Lee S, Valim C, Zhou J, Kim S, Modi BP, Arsenault DA, Strijbosch RA, Lopes S, Duggan C, Puder M. Safety and efficacy of a fish-oil-based fat emulsion in the treatment of parenteral nutrition-associated liver disease. Pediatrics. 2008 Mar;121(3):e678-86. doi: 10.1542/peds.2007-2248.

  PMID: 18310188 BACKGROUND

• Diamond IR, Sterescu A, Pencharz PB, Wales PW. The rationale for the use of parenteral omega-3 lipids in children with short bowel syndrome and liver disease. Pediatr Surg Int. 2008 Jul;24(7):773-8. doi: 10.1007/s00383-008-2174-0. Epub 2008 May 27.

  PMID: 18504595 BACKGROUND

• de Meijer VE, Gura KM, Le HD, Meisel JA, Puder M. Fish oil-based lipid emulsions prevent and reverse parenteral nutrition-associated liver disease: the Boston experience. JPEN J Parenter Enteral Nutr. 2009 Sep-Oct;33(5):541-7. doi: 10.1177/0148607109332773. Epub 2009 Jul 1.

  PMID: 19571170 BACKGROUND

• Park HW, Lee NM, Kim JH, Kim KS, Kim SN. Parenteral fish oil-containing lipid emulsions may reverse parenteral nutrition-associated cholestasis in neonates: a systematic review and meta-analysis. J Nutr. 2015 Feb;145(2):277-83. doi: 10.3945/jn.114.204974. Epub 2014 Dec 17.

  PMID: 25644348 BACKGROUND

MeSH Terms

Conditions

Cholestasis; Liver Diseases; Premature Birth; Intestinal Atresia; Intestinal Failure

Interventions

fish oil triglycerides

Condition Hierarchy (Ancestors)

Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Obstetric Labor, Premature; Obstetric Labor Complications; Pregnancy Complications; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Digestive System Abnormalities; Intestinal Diseases; Gastrointestinal Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: expanded access
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor of Pediatrics

Study Record Dates

• First Submitted: March 2, 2017
• First Posted: March 7, 2017
• Last Updated: December 6, 2018

Record last verified: 2018-12

Locations

US (1)