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
Study 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
Sex
ALL
Min age
1 Day
Max age
17 Years
Healthy volunteers
No
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.
Primary outcome measure(s)
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)
Trial sites (10)
Facility
City
Region
Status
Memorial Health Service
Fountain Valley
California
Recruiting
University of California Los Angeles
Los Angeles
California
Recruiting
The University of Chicago
Chicago
Illinois
Recruiting
Children's Hospital Corporation d/b/a Boston Children's Hospital
Boston
Massachusetts
Recruiting
Children's Hospital Medical Center
Cincinnati
Ohio
Recruiting
Board of Regents of the University of Oklahoma Health Sciences Center
Oklahoma City
Oklahoma
Recruiting
Vanderbilt University Medical Center
Nashville
Tennessee
Recruiting
Baylor College of Medicine Houston
Houston
Texas
Recruiting
The University of Texas Health Science Center at San Antonio
San Antonio
Texas
Recruiting
Seattle Children's Hospital d/b/a Seattle Children's Research Institute
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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