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NCT06274788Recruiting

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

Fresenius Kabi

Start Date

12/15/2024

Completion Date

3/1/2027

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

Eligibility Criteria

Age Range: No minimum to 17 years

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.

Interventions

DRUG

Omegaven® (fish oil triglycerides) Injectable Emulsion

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Conditions

Parenteral Nutrition Associated Liver Disease (PNALD)Essential Fatty Acid DeficiencyMalnutritionPediatric ALL

Locations

Memorial Health Service

Fountain Valley, California 92708

United States

University of California Los Angeles

Los Angeles, California 10911

United States

The University of Chicago

Chicago, Illinois 60637

United States

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

Boston, Massachusetts 02115

United States

Children's Hospital Medical Center

Cincinnati, Ohio 45229

United States

Board of Regents of the University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma 73104

United States

Vanderbilt University Medical Center

Nashville, Tennessee 37232

United States

Baylor College of Medicine Houston

Houston, Texas 77030

United States

The University of Texas Health Science Center at San Antonio

San Antonio, Texas 78229-3901

United States

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

Seattle, Washington 98105

United States