Omegaven® in the Treatment of Parenteral Nutrition (PN) Induced Liver Injury

NCT01845116 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: Omegaven 04-10-18B
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine whether the omega-3 fatty acid emulsion (Omegaven®), when used in place of the conventional soy-based fat emulsion (Intralipid), is effective in treating parenteral nutrition associated liver disease (PNALD) in children. The study hypothesis is that Omegaven® can be safely provided to children who are dependent on parenteral nutrition and have PNALD, and can reverse or prevent progression of PNALD until the child can take adequate nutrition by mouth.

Conditions

Total Parenteral Nutrition-induced Cholestasis

Keywords

PNALD; Cholestasis; Omegaven®; Short Bowel Syndrome

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Normalization of Direct Bilirubin

  Normalization of direct bilirubin is defined as less than 0.4mg/dL by 9 months. This relates to the efficacy of parenteral administration of fish oil derived fat emulsion (Omegaven®) to reverse established parenteral nutrition associated liver disease.

  Month 9

Secondary Outcomes (3)

• Number of Participants With Normal Essential Fatty Acid (EFA) Profiles.

  Months 1 through 9

• Number of Participants With Triglyceride Levels > 400 mg/dL

  baseline data and then weekly and monthly evaluations, for an average of 9 months

• Number of Participants With Unexpected Bleeding or Coagulopathies

  Month 6

Study Arms (1)

Single Arm

OTHER

Single Omegaven® Intervention Arm

Drug: Omegaven®

Interventions

Omegaven® DRUG

10% Omegaven® initiated at a starting dose of 0.5g/kg/day for two days and then advancing to the goal dose of 1g/kg/day, IV (in the vein) until the patient no longer requires parenteral nutrition or until participation in the study is terminated.

Also known as: IV Fish Oil Based Lipid Emulsion

Single Arm

Eligibility Criteria

• Age: 4 Weeks - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Children 0-18 years of age
• Patients will be PN-dependent and expected to continue PN for at least 30 days
• Patients considered eligible for study participation must have PN-associated liver diseases . Other causes of liver disease (i.e., biliary atresia, galactosemia, alpha-1 antitrypsin deficiency) will be excluded. A liver biopsy is not necessary for treatment
• Direct bilirubin \> 2.0 mg/dl
• Signed patient informed consent
• Signed patient assent where applicable.

You may not qualify if:

• Pregnancy
• Other causes of chronic liver disease (cystic fibrosis, biliary atresia, alpha-1 antitrypsin deficiency)
• Signs of advanced liver disease including cirrhosis on biopsy, varices, ascites
• The patient is allergic to eggs/shellfish
• The patient has a severe hemorrhagic disorder
• The patient is enrolled in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team)
• The parent or guardian or child unwilling to provide consent or assent.

Sponsors & Collaborators

• Wake Forest University Health Sciences: lead

Study Sites (1)

Levine Children's Hospital at Carolinas HealthCare System

Charlotte, North Carolina, 28232-2861, United States

Location

Related Publications (3)

• Puder M, Valim C, Meisel JA, Le HD, de Meijer VE, Robinson EM, Zhou J, Duggan C, Gura KM. Parenteral fish oil improves outcomes in patients with parenteral nutrition-associated liver injury. Ann Surg. 2009 Sep;250(3):395-402. doi: 10.1097/SLA.0b013e3181b36657.

  PMID: 19661785 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

• 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

MeSH Terms

Conditions

Cholestasis; Short Bowel Syndrome

Interventions

fish oil triglycerides

Condition Hierarchy (Ancestors)

Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Limitations and Caveats

The last patient enrolled in the study was on 11/8/2016. Due to decreased enrollment permission was granted by the Atrium Health IRB to officially close the study to new enrollees on May 31, 2019. The last patient enrolled is now 5 years removed from enrollment date.

Results Point of Contact

• Title: Ricardo A. Caicedo, MD
• Organization: Atrium Health Levine Children's Hospital

ricardo.caicedo@atriumhealth.org

704-381-8840

Study Officials

• Ricardo A. Caicedo, MD

  Levine Children's Hospital at Carolinas HealthCare System

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 29, 2013
• First Posted: May 3, 2013
• Study Start: July 1, 2010
• Primary Completion: July 1, 2020
• Study Completion: July 1, 2020
• Last Updated: November 23, 2022
• Results First Posted: November 23, 2022

Record last verified: 2022-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)