Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment

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 will demonstrate safety in pediatric patients with Parenteral Nutrition-Associated Cholestasis treated with Omegaven®, which is indicated as a source of calories and fatty acids in this patient population

Study Overview

• Status: Recruiting
• Conditions: Malnutrition; Pediatric ALL; Essential Fatty Acid Deficiency; Parenteral Nutrition Associated Liver Disease (PNALD)
• Intervention / Treatment: Drug: Omegaven® (fish oil triglycerides) Injectable Emulsion

• Study Type: Observational
• Enrollment (Estimated): 40
• Phase: Phase 4

Contacts and Locations

• Study Contact: Name: Lohse Jean-Marc, PhD; Phone Number: +49 173 5420453; Email: Jean-Marc.Lohse@fresenius-kabi.com
• Study Contact Backup: Name: Niess Ulf, PhD; Phone Number: M +49 173 5439924; Email: ulf.niess@fresenius-kabi.com

Study Locations

• United States
  • California
    • Fountain Valley, California, United States, 92708
      • Recruiting
      • Memorial Health Service
    • Los Angeles, California, United States, 10911
      • Recruiting
      • University of California Los Angeles
  • Illinois
    • Chicago, Illinois, United States, 60637
      • Recruiting
      • The University of Chicago
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Recruiting
      • Children's Hospital Corporation d/b/a Boston Children's Hospital
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Recruiting
      • Children's Hospital Medical Center
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • Recruiting
      • Board of Regents of the University of Oklahoma Health Sciences Center
  • Tennessee
    • Nashville, Tennessee, United States, 37232
      • Recruiting
      • Vanderbilt University Medical Center
  • Texas
    • Houston, Texas, United States, 77030
      • Recruiting
      • Baylor College of Medicine Houston
    • San Antonio, Texas, United States, 78229-3901
      • Recruiting
      • The University of Texas Health Science Center at San Antonio
  • Washington
    • Seattle, Washington, United States, 98105
      • Recruiting
      • Seattle Children's Hospital d/b/a Seattle Children's Research Institute

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Male and female pediatric patients with PNAC and expected requirement of Omegaven for at least 8 weeks will be enrolled at multiple sites; PNAC is defined as being PN-dependent with a DBil level ≥ 2.0 mg/dL and no other known cause of liver dysfunction.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

Single arm OMEGAVEN® (fish oil triglycerides; injectable emulsion); The dose of investigational drug (study treatment), as well as all other components of the overall nutritional regimen is solely at the discretion of the Investigator. It is assumed the Investigator will use sound medical judgement, follow institutional standards of care regarding the nutrition provided to each patient, and review applicable prescribing information indicating the maximum and recommended dose of Omegaven of 1 g/kg/day infused intravenously over 8 to 24 hours as long as the infusion rate does not exceed 1.5 mL/kg/hour.

Drug: Omegaven® (fish oil triglycerides) Injectable Emulsion; Pediatric patients Pediatric patients with new-onset PNAC Drug: Omegaven® (fish oil triglycerides) Injectable Emulsion Dose, frequency and duration is a decision of the Investigator

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of essential fatty acid deficiency (EFAD)	EFAD as defined by the triene:tetraene (T:T) ratio; severity of EFAD will be graded and analysed based on T:T ratio (suspected ≥ 0.05, moderate ≥ 0.20, and severe ≥ 0.40)	Up to week 56

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of clinical EFAD	Clinical EFAD is defined by clinical symptoms (like dry, scaly rash, hair loss, hair depigmentation, poor wound healing, growth restriction, and increased susceptibility to infection) and T:T ratio ≥ 0.083	Up to week 56
Time from treatment start to EFAD diagnosis	Time from treatment start to EFAD diagnosis	Up to week 56
Incidence of adverse events (AEs)/serious adverse events (SAEs)	The incidence of AEs/SAEs considered by the Investigator to be related to study treatment	Up to week 56
Routine laboratory tests: Direct or conjugated bilirubin		Up to week 56
Routine laboratory tests: triglycerides		Up to week 56
Fatty acid profiles: α-linolenic acid		Up to week 56
Fatty acid profiles: linoleic acid		Up to week 56
Fatty acid profiles: arachidonic acid		Up to week 56
Fatty acid profiles: mead acid		Up to week 56
Fatty acid profiles: oleic acid		Up to week 56
Fatty acid profiles: docosahexaenoic acid (DHA)		Up to week 56
Fatty acid profiles: eicosapentaenoic acid (EPA)		Up to week 56
Anthropometric measures: body weight		Up to week 56
Anthropometric measures: body height/length		Up to week 56
Anthropometric measures: head circumference		Up to week 56

Collaborators and Investigators

• Sponsor: Fresenius Kabi

Study record dates

Study Major Dates

• Study Start (Actual): December 15, 2024
• Primary Completion (Estimated): March 1, 2026
• Study Completion (Estimated): March 1, 2027

Study Registration Dates

• First Submitted: January 26, 2024
• First Submitted That Met QC Criteria: February 15, 2024
• First Posted (Actual): February 23, 2024

Study Record Updates

• Last Update Posted (Estimated): September 5, 2025
• Last Update Submitted That Met QC Criteria: August 28, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nutrition Disorders; Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Leukemia, Lymphoid; Leukemia; Nutritional and Metabolic Diseases; Hemic and Lymphatic Diseases; Malnutrition; Precursor Cell Lymphoblastic Leukemia-Lymphoma; fish oil triglycerides
• Other Study ID Numbers: OMEG-054-CP4

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No