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

Official title

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

Overview

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

Interventions

• Drug 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

Primary outcome measures

• Incidence of essential fatty acid deficiency (EFAD) [Time frame: Up to week 56]

Secondary outcome measures (12)

• Incidence of clinical EFAD [Time frame: Up to week 56]
• Time from treatment start to EFAD diagnosis [Time frame: Up to week 56]
• Incidence of adverse events (AEs)/serious adverse events (SAEs) [Time frame: Up to week 56]
• Routine laboratory tests: Direct or conjugated bilirubin [Time frame: Up to week 56]
• Routine laboratory tests: triglycerides [Time frame: Up to week 56]
• Fatty acid profiles: α-linolenic acid [Time frame: Up to week 56]
• Fatty acid profiles: linoleic acid [Time frame: Up to week 56]
• Fatty acid profiles: arachidonic acid [Time frame: Up to week 56]
• Fatty acid profiles: mead acid [Time frame: Up to week 56]
• Fatty acid profiles: oleic acid [Time frame: Up to week 56]
• Fatty acid profiles: docosahexaenoic acid (DHA) [Time frame: Up to week 56]
• Fatty acid profiles: eicosapentaenoic acid (EPA) [Time frame: Up to week 56]

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.
• 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 a record of EFAD before inclusion in the study
• 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Other

Study locations

United States · 10 centers

• Memorial Health Service — Fountain Valley
• University of California Los Angeles — Los Angeles
• The University of Chicago — Chicago
• Children's Hospital Corporation d/b/a Boston Children's Hospital — Boston
• Children's Hospital Medical Center — Cincinnati
• Board of Regents of the University of Oklahoma Health Sciences Center — Oklahoma City
• Vanderbilt University Medical Center — Nashville
• Baylor College of Medicine Houston — Houston
• … and 2 more centers

Identifiers

NCT: NCT06274788 · OMEG-054-CP4

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗