Safety of Omegaven in Pediatric Patients With Parenteral Nutrition-Associated Cholestasis (PNAC)

NCT04807478 | Withdrawn FDA Drug

• 1 other identifier: OMEG-038-CP4
• Study Type: observational
• Target: N/A
• Locations: 0 countries
• Sites: N/A

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

Conditions

Parenteral Nutrition Associated Liver Disease; Essential Fatty Acid Deficiency; Neurocognitive Deficit; Malnutrition; Pediatric ALL

Outcome Measures

Primary Outcomes (1)

• Incidence of:

  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.

  Through study completion, a maximum of 6 years

Other Outcomes (1)

• Adverse events (AEs)/serious adverse events (SAEs)

  Through study completion, a maximum of 6 years

Study Arms (1)

Pediatric patients

Pediatric patients with new-onset PNAC

Drug: Omegaven® (fish oil triglycerides) Injectable Emulsion

Interventions

Omegaven® (fish oil triglycerides) Injectable Emulsion DRUG

Dose, frequency and duration is a decision of the Investigator

Pediatric patients

Eligibility Criteria

• Age: 1 Day+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Male and female pediatric patients \< 6 months old (corrected age) with new-onset PNAC (diagnosed within the past 21 days) will be enrolled at multiple sites; PNAC is defined as being PN-dependent with a DBil level ≥ 2.0 mg/dL and no other known cause of liver dysfunction.

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Fresenius Kabi: lead

MeSH Terms

Conditions

Malnutrition; Precursor Cell Lymphoblastic Leukemia-Lymphoma

Interventions

fish oil triglycerides

Condition Hierarchy (Ancestors)

Nutrition Disorders; Nutritional and Metabolic Diseases; Leukemia, Lymphoid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 10, 2021
• First Posted: March 19, 2021
• Study Start: April 1, 2022
• Primary Completion (Estimated): May 1, 2028
• Study Completion (Estimated): May 1, 2029
• Last Updated: August 4, 2022

Record last verified: 2022-08