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

NCT06274788 | Recruiting FDA Drug

• 1 other identifier: OMEG-054-CP4
• Study Type: observational
• Target: 40
• Locations: 1 country
• Sites: 10

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

Conditions

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (15)

• Incidence of clinical EFAD

  Up to week 56

• Time from treatment start to EFAD diagnosis

  Up to week 56

• Incidence of adverse events (AEs)/serious adverse events (SAEs)

  Up to week 56

• Routine laboratory tests: Direct or conjugated bilirubin

  Up to week 56

• Routine laboratory tests: triglycerides

  Up to week 56

Study Arms (1)

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

Interventions

Omegaven® (fish oil triglycerides) Injectable Emulsion 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

Single arm OMEGAVEN® (fish oil triglycerides; injectable emulsion)

Eligibility Criteria

• Age: 1 Day - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• 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.

You may qualify if:

• Patient's parent(s) or legal guardian(s) has provided a signed and dated Informed Consent Form (ICF).
• 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.
• Patient has oral or enteral feeding intolerance or at least one gastrointestinal disorder requiring PN.

You may not qualify if:

• 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.
• Patient has known cirrhosis (liver biopsy is not required under this protocol).
• Patient has been previously diagnosed with, or has prior evidence of, portal vein thrombosis.
• Patient has previously received a liver-only or liver-inclusive transplant.
• Patient has hemodynamic instability due to any major cardiac anomaly.
• Patient has a major life-threatening disease (e.g., sepsis requiring high-dose vasopressors, acute respiratory distress syndrome, veno-occlusive disease, cancer).
• 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.
• Patient has renal failure and requires renal replacement therapy.
• Patient has a severe hemorrhagic disorder.
• Patient has severe hyperlipidemia or a severe disorder of lipid metabolism characterized by hypertriglyceridemia (i.e., serum triglyceride level \> 1,000 mg/dL).
• Patient has been diagnosed with or is suspected to have an inborn error of metabolism.
• Patient has a known hypersensitivity to fish or egg protein or to any of the active ingredients or excipients of Omegaven.
• Patient is subject to treatment limitation.
• Patient is enrolled in any other study with an investigational medicinal product during the course of the current study.

Sponsors & Collaborators

• Fresenius Kabi: lead

Study Sites (10)

Memorial Health Service

Fountain Valley, California, 92708, United States

RECRUITING

University of California Los Angeles

Los Angeles, California, 10911, United States

RECRUITING

The University of Chicago

Chicago, Illinois, 60637, United States

RECRUITING

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

Boston, Massachusetts, 02115, United States

RECRUITING

Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

RECRUITING

Board of Regents of the University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

RECRUITING

Baylor College of Medicine Houston

Houston, Texas, 77030, United States

RECRUITING

The University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229-3901, United States

RECRUITING

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

Seattle, Washington, 98105, United States

RECRUITING

MeSH Terms

Conditions

Malnutrition; Precursor Cell Lymphoblastic Leukemia-Lymphoma

Interventions

fish oil triglycerides

Condition Hierarchy (Ancestors)

Nutrition Disorders; Nutritional and Metabolic Diseases; Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Central Study Contacts

Niess Ulf, PhD

CONTACT

M +49 173 5439924; ulf.niess@fresenius-kabi.com

Lohse Jean-Marc, PhD

CONTACT

+49 173 5420453; Jean-Marc.Lohse@fresenius-kabi.com

Study Design

• Study Type: observational
• Observational Model: OTHER
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2024
• First Posted: February 23, 2024
• Study Start: December 15, 2024
• Primary Completion: March 1, 2026
• Study Completion (Estimated): March 1, 2027
• Last Updated: September 5, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (10)