A Multicenter, Prospective, Controlled, Longitudinal-Cohort Study Assessing the Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)
Overview
- Phase
- N/A
- Intervention
- Omegaven® (fish oil triglycerides) Injectable Emulsion
- Conditions
- Parenteral Nutrition Associated Liver Disease
- Sponsor
- Fresenius Kabi
- Primary Endpoint
- Incidence of:
- Status
- Withdrawn
- Last Updated
- 3 years ago
Overview
Brief Summary
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
Detailed 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.
Investigators
Eligibility Criteria
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.
Arms & Interventions
Pediatric patients
Pediatric patients with new-onset PNAC
Intervention: Omegaven® (fish oil triglycerides) Injectable Emulsion
Outcomes
Primary Outcomes
Incidence of:
Time Frame: Through study completion, a maximum of 6 years
1. Incidence of EFAD 2. Incidence of serious bleeding events 3. Incidence of life-threatening pericardial effusion events 4. Incidence of life-threatening pleural effusion events 5. Severity of any neurodevelopmental delays.