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

NCT ID: NCT06274788

Last Updated: 2025-09-05

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

40 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-12-15

Study Completion Date

2027-03-01

Brief Summary

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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

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Conditions

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Parenteral Nutrition Associated Liver Disease (PNALD) Essential Fatty Acid Deficiency Malnutrition Pediatric ALL

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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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.

Omegaven® (fish oil triglycerides) Injectable Emulsion

Intervention Type DRUG

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

Interventions

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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

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

1 Day

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Fresenius Kabi

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Memorial Health Service

Fountain Valley, California, United States

Site Status RECRUITING

University of California Los Angeles

Los Angeles, California, United States

Site Status RECRUITING

The University of Chicago

Chicago, Illinois, United States

Site Status RECRUITING

Children's Hospital Corporation d/b/a Boston Children's Hospital

Boston, Massachusetts, United States

Site Status RECRUITING

Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status RECRUITING

Board of Regents of the University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, United States

Site Status RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status RECRUITING

Baylor College of Medicine Houston

Houston, Texas, United States

Site Status RECRUITING

The University of Texas Health Science Center at San Antonio

San Antonio, Texas, United States

Site Status RECRUITING

Seattle Children's Hospital d/b/a Seattle Children's Research Institute

Seattle, Washington, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Niess Ulf, PhD

Role: CONTACT

M +49 173 5439924

Lohse Jean-Marc, PhD

Role: CONTACT

+49 173 5420453

Other Identifiers

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OMEG-054-CP4

Identifier Type: -

Identifier Source: org_study_id

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