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

Fresenius Kabi10 sites in 1 country40 target enrollmentDecember 15, 2024

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

InterventionsOmegaven® (fish oil triglycerides) Injectable Emulsion

DrugsOmegaven® (fish oil triglycerides) Injectable Emulsion

Overview

Phase: Phase 4
Intervention: Omegaven® (fish oil triglycerides) Injectable Emulsion
Conditions: Parenteral Nutrition Associated Liver Disease (PNALD)
Sponsor: Fresenius Kabi
Enrollment: 40
Locations: 10
Primary Endpoint: Incidence of essential fatty acid deficiency (EFAD)
Status: Recruiting
Last Updated: 7 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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

Registry

clinicaltrials.gov

Start Date

December 15, 2024

End Date

March 1, 2027

Last Updated

7 months ago

Study Type

Observational

Sex

All

Investigators

Sponsor

Fresenius Kabi

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•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.

Exclusion Criteria

•Patient has received Omegaven within four weeks before inclusion in the study
•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.

Arms & Interventions

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.

Intervention: Omegaven® (fish oil triglycerides) Injectable Emulsion

Outcomes

Primary Outcomes

Incidence of essential fatty acid deficiency (EFAD)

Time Frame: 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)