Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)
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About
This study will demonstrate Short-, mid-, and long-term 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
Full description
Prospective, controlled, longitudinal-cohort phase IV study to assess safety of Omegaven in Pediatric Patients with PNAC, in order to demonstrate short-, mid-, and long-term safety with respect to EFAD, serious bleeding events, life-threatening pleural and pericardial effusions, and neurocognitive development. Pediatric patients with PNAC receiving Omegaven as part of their routine nutritional management will be compared to those treated with another FDA-approved ILE for pediatric patients. Patients who are switched to a non-FDA-approved ILE or to Omegaven will be analyzed separately. Use of ILE will be as indicated by their treating physician, ideally for ≥ 1 year. Dosing modalities are to be taken from the pertinent prescribing information.
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Inclusion criteria
- Patient's parent(s) or legal guardian(s) has provided a signed and dated Informed Consent Form (ICF).
- Patient is PN-dependent and within the past 21 days 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.
- Patient has feeding intolerance or at least one gastrointestinal disorder requiring PN.
- Patient is < 6 months corrected age (expected time of delivery to time of screening).
Exclusion criteria
- 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 known portal vein thrombosis (imaging studies are not required under this protocol).
- Patient has previously received a liver-only or liver-inclusive transplant.
- Patient has a major cardiac anomaly with hemodynamic instability.
- 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 dialysis.
- Patient has a severe hemorrhagic disorder.
- Patient has an INR > 2.0.
- 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 a previous T:T ratio ≥ 0.2 or had a previous diagnosis of EFAD.
- Patient has a central nervous system anomaly (e.g., anencephaly) or any injury (e.g., grade 3 or 4 intraventricular hemorrhage, moderate to severe hypoxic ischemic encephalopathy) that will affect neurodevelopment.
- Patient has been diagnosed with or is suspected to have a genetic disorder known to be associated with neurodevelopmental impairment (e.g., trisomy 21, 18, 13).
- 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's medical care has been deemed futile by the medical team.
- Patient is enrolled in any other study with an investigational medicinal product.
Trial design
0 participants in 1 patient group
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Data sourced from clinicaltrials.gov
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