Evaluating the safety of Omegaven in children with liver disease from parenteral nutrition

Title: A Single-arm, Open-label, Prospective, Multicenter Safety Study to Evaluate the Occurrence of Essential Fatty Acid Deficiency (EFAD) in Pediatric Patients With Parenteral Nutrition-associated Cholestasis (PNAC) Who Require More Than Eight Weeks of Omegaven Treatment

This study is testing whether Omegaven, a fish oil treatment, is safe for children with liver disease caused by long-term feeding through an IV.

Quick facts

• Study type: Observational
• Enrollment: 40 (estimated)
• Ages: 1 Day to 17 Years
• Sex: All
• Sponsor: Fresenius Kabi (Industry-sponsored)
• Locations: 10 sites (Fountain Valley, California and 9 other locations)
• Trial ID: NCT06274788 on ClinicalTrials.gov

What this trial studies

This observational study aims to assess the safety of Omegaven®, a fish oil triglyceride injectable emulsion, in pediatric patients diagnosed with Parenteral Nutrition-Associated Cholestasis (PNAC). The study will include children under 18 years who require Omegaven treatment for more than eight weeks due to malnutrition or gastrointestinal disorders. Participants will be monitored for the occurrence of essential fatty acid deficiency (EFAD) during their treatment. The study will gather data to ensure the safety of this treatment approach in a vulnerable pediatric population.

Who should consider this trial

Good fit: Ideal candidates are pediatric patients under 18 years diagnosed with PNAC who require Omegaven treatment for at least eight weeks.

Not a fit: Patients with other known causes of chronic liver disease or those who have received Omegaven within four weeks prior to the study will not benefit.

Why it matters

Potential benefit: If successful, this study could provide a safer treatment option for pediatric patients suffering from liver disease associated with parenteral nutrition.

How similar studies have performed: While this approach is being evaluated for safety, similar studies using Omegaven in pediatric populations have shown promising results.

Eligibility criteria

Inclusion Criteria:

1. Patient's parent(s) or legal guardian(s) has provided a signed and dated Informed Consent Form (ICF).
2. Pediatric patient (\<18 years) has been diagnosed with PNAC, defined as direct or conjugated bilirubin (DBil) ≥ 2.0 mg/dL with no other known cause of liver dysfunction at the time of enrollment and is expected to require Omegaven treatment for at least eight weeks.
3. Patient has oral or enteral feeding intolerance or at least one gastrointestinal disorder requiring PN.

Exclusion Criteria:

1. Patient has received Omegaven within four weeks before inclusion in the study
2. Patient has any other known cause of chronic liver disease such as hepatitis C, cystic fibrosis, biliary atresia, alpha-1-antitrypsin deficiency, passive hepatic congestion due to heart failure, etc.
3. Patient has known cirrhosis (liver biopsy is not required under this protocol).
4. Patient has been previously diagnosed with, or has prior evidence of, portal vein thrombosis.
5. Patient has previously received a liver-only or liver-inclusive transplant.
6. Patient has hemodynamic instability due to any major cardiac anomaly.
7. Patient has a major life-threatening disease (e.g., sepsis requiring high-dose vasopressors, acute respiratory distress syndrome, veno-occlusive disease, cancer).
8. Patient has multi-organ failure, septic shock, hypotension requiring pressor therapy, persistent pulmonary hypertension requiring inhaled nitric oxides, or requires extracorporeal membrane oxygenation (ECMO) or similar intervention.
9. Patient has renal failure and requires renal replacement therapy.
10. Patient has a severe hemorrhagic disorder.
11. Patient has severe hyperlipidemia or a severe disorder of lipid metabolism characterized by hypertriglyceridemia (i.e., serum triglyceride level \> 1,000 mg/dL).
12. Patient has a record of EFAD before inclusion in the study
13. Patient has been diagnosed with or is suspected to have an inborn error of metabolism.
14. Patient has a known hypersensitivity to fish or egg protein or to any of the active ingredients or excipients of Omegaven.
15. Patient is subject to treatment limitation.
16. Patient is enrolled in any other study with an investigational medicinal product during the course of the current study.

Where this trial is running

Fountain Valley, California and 9 other locations

• Memorial Health Service — Fountain Valley, California, United States (Recruiting)
• University of California Los Angeles — Los Angeles, California, United States (Recruiting)
• The University of Chicago — Chicago, Illinois, United States (Recruiting)
• Children's Hospital Corporation d/b/a Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
• Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
• Board of Regents of the University of Oklahoma Health Sciences Center — Oklahoma City, Oklahoma, United States (Recruiting)
• Vanderbilt University Medical Center — Nashville, Tennessee, United States (Recruiting)
• Baylor College of Medicine Houston — Houston, Texas, United States (Recruiting)
• The University of Texas Health Science Center at San Antonio — San Antonio, Texas, United States (Recruiting)
• Seattle Children's Hospital d/b/a Seattle Children's Research Institute — Seattle, Washington, United States (Recruiting)

Study contacts

• Study coordinator: Niess Ulf, PhD

Conditions Parenteral Nutrition Associated Liver Disease; Essential Fatty Acid Deficiency; Malnutrition; Pediatric ALL