Safety Study to Evaluate the Occurrence of EFAD in Pediatric Patients With PNAC Who Require More Than Eight Weeks of Omegaven Treatment
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
Who can join
As published by the study on ClinicalTrials.gov. "Not stated" means the study hasn't said — ask the study team.
- Age
- 1 Day to 17 Years
- Sex
- All sexes
- Healthy volunteers
- Not accepted
- Compensation
- Not stated
Other requirements are listed under Eligibility below. Only the study team can confirm whether you can join.
What to expect
No one can promise a trial will help you personally — that is what the research is trying to find out.
What participation involves
Not stated in the study description — ask the study team about visits and procedures.
- Compensation
- Not stated
- Study-related exams and procedures are typically provided at no cost. This is how many trials work in general — confirm the details for this study with the study team.
- Participants are monitored closely by the research team throughout the study.
- Some participants may receive access to an investigational treatment, which is still being studied.
- Your participation helps researchers learn whether this approach could help future patients.
About this study
In plain language
This study checks the safety of Omegaven in children under 18 who need it for at least eight weeks. These children have PNAC, a liver problem linked to nutrition given through a vein. The study looks for a lack of essential fats the body needs, called EFAD.
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
Compensation is read from the study's own description and may be missing — always confirm with the study team.
Eligibility
Who can join
- A parent or legal guardian must sign and date a consent form.
- The child must be under 18 and have PNAC, with no other known cause of liver problems. Direct or conjugated bilirubin, a substance measured in blood, must be at least 2.0 mg/dL.
- The child must be expected to need Omegaven for at least eight weeks.
- The child cannot tolerate feeding by mouth or feeding tube, or has a digestive problem that requires nutrition through a vein.
Who can't join
- Received Omegaven in the past four weeks, or had EFAD before joining the study.
- Has another known cause of long-term liver disease, severe liver scarring, a past blood clot in the vein leading to the liver, or a transplant that included the liver.
- Has unstable blood flow due to a major heart defect, or a major life-threatening disease, such as cancer, severe infection, severe lung distress, or blocked small liver veins.
- Has failure of several organs, shock from infection, low blood pressure needing medicine to raise it, high blood pressure in the lungs needing inhaled nitric oxide, or needs an ECMO machine or similar support for the heart and lungs.
- Has kidney failure needing dialysis or similar treatment, or a severe bleeding disorder.
- Has very high blood fat levels or a severe problem processing fats, with triglycerides above 1,000 mg/dL; or has a known or suspected inherited problem processing nutrients.
- Has a known allergy to fish protein, egg protein, or any ingredient in Omegaven.
- Has limits placed on medical treatment, or is taking part in another study of an experimental medicine during this study.
Simplified for readability. The official criteria below are what the study team uses.
Official 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.
Study sites (10)
CA
- Memorial Health Service · Fountain Valley, CA 92708More trials in Fountain Valley
- University of California Los Angeles · Los Angeles, CA 10911More trials in Los Angeles
IL
- The University of Chicago · Chicago, IL 60637More trials in Chicago
MA
- Children's Hospital Corporation d/b/a Boston Children's Hospital · Boston, MA 02115More trials in Boston
OH
- Children's Hospital Medical Center · Cincinnati, OH 45229More trials in Cincinnati
OK
- Board of Regents of the University of Oklahoma Health Sciences Center · Oklahoma City, OK 73104More trials in Oklahoma City
TN
- Vanderbilt University Medical Center · Nashville, TN 37232More trials in Nashville
TX
- Baylor College of Medicine Houston · Houston, TX 77030More trials in Houston
- The University of Texas Health Science Center at San Antonio · San Antonio, TX 78229-3901More trials in San Antonio
WA
- Seattle Children's Hospital d/b/a Seattle Children's Research Institute · Seattle, WA 98105More trials in Seattle