Essential Fatty Acid Deficiency, Malnutrition, Parenteral Nutrition Associated Liver Disease (PNALD), Pediatric ALL
Conditions
Brief 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
Interventions
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
Sponsors
Fresenius Kabi
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
1 Days to 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of clinical EFAD | Up to week 56 | Clinical EFAD is defined by clinical symptoms (like dry, scaly rash, hair loss, hair depigmentation, poor wound healing, growth restriction, and increased susceptibility to infection) and T:T ratio ≥ 0.083 |
| Time from treatment start to EFAD diagnosis | Up to week 56 | Time from treatment start to EFAD diagnosis |
| Incidence of adverse events (AEs)/serious adverse events (SAEs) | Up to week 56 | The incidence of AEs/SAEs considered by the Investigator to be related to study treatment |
| Routine laboratory tests: Direct or conjugated bilirubin | Up to week 56 | — |
| Routine laboratory tests: triglycerides | Up to week 56 | — |
| Fatty acid profiles: α-linolenic acid | Up to week 56 | — |
| Fatty acid profiles: linoleic acid | Up to week 56 | — |
| Fatty acid profiles: arachidonic acid | Up to week 56 | — |
| Fatty acid profiles: mead acid | Up to week 56 | — |
| Fatty acid profiles: oleic acid | Up to week 56 | — |
| Fatty acid profiles: docosahexaenoic acid (DHA) | Up to week 56 | — |
| Fatty acid profiles: eicosapentaenoic acid (EPA) | Up to week 56 | — |
| Anthropometric measures: body weight | Up to week 56 | — |
| Anthropometric measures: body height/length | Up to week 56 | — |
| Anthropometric measures: head circumference | Up to week 56 | — |
Countries
United States
Contacts
CONTACTNiess Ulf, PhD
CONTACTLohse Jean-Marc, PhD
Outcome results
None listed