Clinical trial NCT06274788: 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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Parenteral Nutrition Associated Liver Disease (PNALD)
Essential Fatty Acid Deficiency
Malnutrition
Pediatric ALL
Lead sponsor: Fresenius Kabi
Sponsor class: INDUSTRY
First posted: 2024-02-23
Enrollment: 40
Sex: ALL
Minimum age (years): 1
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Male and female pediatric patients with PNAC and expected requirement of Omegaven for at least 8 weeks 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.

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


Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of essential fatty acid deficiency (EFAD)
timeFrame: Up to week 56
Secondary outcomes:
measure: Incidence of clinical EFAD
timeFrame: Up to week 56
measure: Time from treatment start to EFAD diagnosis
timeFrame: Up to week 56
measure: Incidence of adverse events (AEs)/serious adverse events (SAEs)
timeFrame: Up to week 56
measure: Routine laboratory tests: Direct or conjugated bilirubin
timeFrame: Up to week 56
measure: Routine laboratory tests: triglycerides
timeFrame: Up to week 56
measure: Fatty acid profiles: α-linolenic acid
timeFrame: Up to week 56
measure: Fatty acid profiles: linoleic acid
timeFrame: Up to week 56
measure: Fatty acid profiles: arachidonic acid
timeFrame: Up to week 56
measure: Fatty acid profiles: mead acid
timeFrame: Up to week 56
measure: Fatty acid profiles: oleic acid
timeFrame: Up to week 56
measure: Fatty acid profiles: docosahexaenoic acid (DHA)
timeFrame: Up to week 56
measure: Fatty acid profiles: eicosapentaenoic acid (EPA)
timeFrame: Up to week 56
measure: Anthropometric measures: body weight
timeFrame: Up to week 56
measure: Anthropometric measures: body height/length
timeFrame: Up to week 56
measure: Anthropometric measures: head circumference
timeFrame: Up to week 56
Other outcomes:
Locations:
city: Fountain Valley
country: United States
facility: Memorial Health Service
state: California
city: Los Angeles
country: United States
facility: University of California Los Angeles
state: California
city: Oklahoma City
country: United States
facility: Board of Regents of the University of Oklahoma Health Sciences Center
state: Oklahoma
city: Houston
country: United States
facility: Baylor College of Medicine Houston
state: Texas
city: San Antonio
country: United States
facility: The University of Texas Health Science Center at San Antonio
state: Texas
Study officials:
References: