Omegaven for Compassionate Use in the Treatment of Parenteral Nutrition-Associated Liver Disease (NCT03072667) | Clinical Trial Compass
APPROVED_FOR_MARKETINGNot Applicable
Omegaven for Compassionate Use in the Treatment of Parenteral Nutrition-Associated Liver Disease
United States
Plain-language summary
This is a compassionate use protocol of an investigational new drug (IND). The overall purpose of the treatment is to offer alternative treatment to children who developed parenteral nutrition-associated liver disease (PNALD) and have not responded positively to currently available medical therapies. PNALD develops in newborns dependent on parenteral nutrition (PN) and are unable to tolerate adequate enteral feedings to support fluid and nutritional fluids; although PN is necessary and life sustaining, it can result in severe liver disease.
Who can participate
Age range
1 Year
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age \< 1 years; inpatient use only
. PN dependency (unable to meet nutritional needs solely by enteral nutrition);
. Present PNALD, as evidenced by two consecutive direct bilirubin \> 3.0 mg/dL (at interval of one week);
. Failed standard therapies to prevent the progression of liver disease:
. At the time the diagnosis PNALD is made, the patient is expected to continue PN at least an additional 3 weeks
Exclusion criteria
. Any other cause of chronic liver disease (i.e., hepatitis B or C, cystic fibrosis, biliary atresia, or alpha 1 anti-trypsin deficiency etc.)
. enrollment in any other clinical trial involving an investigational agent (unless approved by the designated physicians on the multidisciplinary team);
. Lack of informed consent;
. Intent to transfer to another healthcare facility
. allergy to any seafood product, egg protein, and/or previous allergy to Omegaven
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. active coagulopathy characterized by ongoing bleeding or by a requirement for clotting factor replacement (e.g. fresh frozen plasma or cryoprecipitate) to maintain homeostasis
. impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis