RECRUITING

Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment

Conditions

Parenteral Nutrition Associated Liver Disease (PNALD)Essential Fatty Acid Deficiency Malnutrition Pediatric ALL

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

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

Quick Facts

Study Start:2024-12-15

Study Completion:2027-03-01

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06274788

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:1 Day to 17 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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.	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.

Inclusion Criteria

Exclusion 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.

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.

Contacts and Locations

Study Contact

Schneider-Schranz Cornelia, PhD

CONTACT

+491737335940

cornelia.schneider-schranz@fresenius-kabi.com

Lohse Jean-Marc, PhD

CONTACT

+49 173 5420453

Jean-Marc.Lohse@fresenius-kabi.com

Study Locations (Sites)

Memorial Health Service

Fountain Valley, California, 92708

United States

Board of Regents of the University of Oklahoma Health Sciences Center

Oklahoma City, Oklahoma, 73104

United States

Baylor College of Medicine Houston

Houston, Texas, 77030

United States

The University of Texas Health Science Center at San Antonio

San Antonio, Texas, 78229-3901

United States

Collaborators and Investigators

Sponsor: Fresenius Kabi

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-12-15

Study Completion Date2027-03-01

Study Record Updates

Study Start Date2024-12-15

Study Completion Date2027-03-01

Terms related to this study

Additional Relevant MeSH Terms

Parenteral Nutrition Associated Liver Disease (PNALD)
Essential Fatty Acid Deficiency
Malnutrition
Pediatric ALL