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

Phase 4

Recruiting

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

• Registration Number: NCT06274788
• Lead Sponsor: Fresenius Kabi

Brief Summary

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-01-26
• Study First Submitted QC: 2024-02-15
• Study First Posted: 2024-02-23
• Study Start: 2024-12-15
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-26
• Last Update Posted: 2025-02-28
• Primary Completion: 2026-03-01
• Study Completion: 2027-03-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Single arm OMEGAVEN® (fish oil triglycerides; injectable emulsion)	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.

Primary Outcome Measures

Name	Time	Method
Incidence of essential fatty acid deficiency (EFAD)	Up to week 56	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)

Secondary Outcome Measures

Name	Time	Method
Time from treatment start to EFAD diagnosis	Up to week 56	Time from treatment start to EFAD diagnosis
Routine laboratory tests: Direct or conjugated bilirubin	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
Incidence of clinical EFAD	Up to week 56	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
Fatty acid profiles: α-linolenic acid	Up to week 56
Anthropometric measures: head circumference	Up to week 56
Incidence of adverse events (AEs)/serious adverse events (SAEs)	Up to week 56	The incidence of AEs/SAEs considered by the Investigator to be related to study treatment
Routine laboratory tests: triglycerides	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

Trial Locations

Locations (5)

University of California Los Angeles; 🇺🇸 Los Angeles, California, United States

Memorial Health Service; 🇺🇸 Fountain Valley, California, United States

Board of Regents of the University of Oklahoma Health Sciences Center; 🇺🇸 Oklahoma City, Oklahoma, United States

Baylor College of Medicine Houston; 🇺🇸 Houston, Texas, United States

The University of Texas Health Science Center at San Antonio; 🇺🇸 San Antonio, Texas, United States