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RecruitingObservational

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

NCT ID: NCT06274788Sponsor: Fresenius KabiLast updated: 2025-09-05

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

Arms & interventions

  • DrugOmegaven® (fish oil triglycerides) Injectable Emulsion

    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

Outcome measures

Primary

  • Incidence of essential fatty acid deficiency (EFAD)

    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)

    Time frame: Up to week 56

Secondary

  • Incidence of clinical EFAD

    Time frame: Up to week 56

  • Time from treatment start to EFAD diagnosis

    Time frame: Up to week 56

  • Incidence of adverse events (AEs)/serious adverse events (SAEs)

    Time frame: Up to week 56

  • Routine laboratory tests: Direct or conjugated bilirubin

    Time frame: Up to week 56

  • Routine laboratory tests: triglycerides

    Time frame: Up to week 56

  • Fatty acid profiles: α-linolenic acid

    Time frame: Up to week 56

  • Fatty acid profiles: linoleic acid

    Time frame: Up to week 56

  • Fatty acid profiles: arachidonic acid

    Time frame: Up to week 56

  • Fatty acid profiles: mead acid

    Time frame: Up to week 56

  • Fatty acid profiles: oleic acid

    Time frame: Up to week 56

  • Fatty acid profiles: docosahexaenoic acid (DHA)

    Time frame: Up to week 56

  • Fatty acid profiles: eicosapentaenoic acid (EPA)

    Time frame: Up to week 56

  • Anthropometric measures: body weight

    Time frame: Up to week 56

  • Anthropometric measures: body height/length

    Time frame: Up to week 56

  • Anthropometric measures: head circumference

    Time frame: Up to week 56

Eligibility criteria

Sex: AllAge: 1 Day to 17 YearsHealthy 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.

Study locations (10)

Memorial Health Service

Fountain Valley, California, 92708

Recruiting

University of California Los Angeles

Los Angeles, California, 10911

Recruiting

The University of Chicago

Chicago, Illinois, 60637

Recruiting

Children's Hospital Corporation d/b/a Boston Children's Hospital

Boston, Massachusetts, 02115

Recruiting

Children's Hospital Medical Center

Cincinnati, Ohio, 45229

Recruiting

Board of Regents of the University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104

Recruiting

Vanderbilt University Medical Center

Nashville, Tennessee, 37232

Recruiting

Baylor College of Medicine Houston

Houston, Texas, 77030

Recruiting

The University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229-3901

Recruiting

Seattle Children's Hospital d/b/a Seattle Children's Research Institute

Seattle, Washington, 98105

Recruiting