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Omegaven Protocol:Intermediate Size Patient Population

Primary Purpose

Parenteral Nutrition Associated Liver Disease

Status
Approved for marketing
Phase
Locations
Study Type
Expanded Access
Intervention
Omegaven
Sponsored by
DHR Health Institute for Research and Development
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an expanded access trial for Parenteral Nutrition Associated Liver Disease

Eligibility Criteria

22 Weeks - 40 Weeks (Child)All Sexes

Inclusion Criteria:

- 1. Inborn and transferred newborns born between the gestational age of 22-40 weeks who develop chronic liver failure, secondary to parenteral nutrition.

2. Patients will be PN dependent (unable to meet nutritional needs soley by enteral nutrition) and are expected to require PN for at least another 30 days 3. Patients must have diagnosis of parenteral nutrition associated liver disease (PNALD) as defined by serum direct bilirubin greater than 2mg/dL on 2 consecutive occasions 4. have had PNALD for at least four weeks prior to planned Omegaven initiation 5. Direct bilirubin > 2.0 mg/dl 6. Signed patient informed consent. 7. The patient must have utilized standard therapies to prevent the progression of his/her liver disease including surgical treatment, cyclic PN, avoiding overfeeding, reduction/removal of copper and manganese from PN, advancement of enteral feeding, and the use of ursodiol (i..e., Actigall®).

Exclusion Criteria:

- 2. Other causes of chronic liver disease (Hepatitis C, Cystic fibrosis, biliary atresia, and alpha 1 anti-trypsin deficiency,) 3. severe and/or unstable concomitant systemic disease such as complex congenital cardiac disease, renal failure, autoimmune disease, sepsis, inborn error of metabolism, genetic liver disease, 4. bleeding disorder; 5. biochemical disturbance with potential of worsening with proposed treatment, e.g. persistent hyperglycemia, hypertriglyceridemia, hypercalcemia.

6. The parent or guardian or child unwilling to provide consent or assent In rare instances, patients diagnosed with PNALD may later be found to have liver disease due to other causes in addition to the use of PN (i.e., inborn errors of metabolism, viral infections ). Such causes may not be known at the time of enrollment and will not preclude them from continuing in the study.

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    June 4, 2018
    Last Updated
    May 7, 2021
    Sponsor
    DHR Health Institute for Research and Development
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    1. Study Identification

    Unique Protocol Identification Number
    NCT03561194
    Brief Title
    Omegaven Protocol:Intermediate Size Patient Population
    Official Title
    Omegaven Protocol:Intermediate Size Patient Population
    Study Type
    Expanded Access

    2. Study Status

    Record Verification Date
    August 2018
    Overall Recruitment Status
    Approved for marketing
    Study Start Date
    undefined (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Responsible Party, by Official Title
    Sponsor
    Name of the Sponsor
    DHR Health Institute for Research and Development

    4. Oversight

    5. Study Description

    Brief Summary
    Children requiring prolonged courses of PN are at risk for developing PN associated liver disease. We hypothesize that although omega-6 fatty acid emulsions prevent fatty acid deficiency, they are not cleared in a manner similar to enteral chylomicrons and therefore accumulate in the liver and resulting in steatotic liver injury. We further hypothesize that a fat emulsion comprised of omega-3 fatty acids (i.e., fish oil) such as Omegaven® would be beneficial in the management of steatotic liver injury by its inhibition of de novo lipogenesis, the reduction of arachidonic acid-derived inflammatory mediators, prevention of essential fatty acid deficiency through the presence of small amounts of arachidonic acid, and improved clearance of lipids from the serum.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Parenteral Nutrition Associated Liver Disease

    7. Study Design

    8. Arms, Groups, and Interventions

    Intervention Type
    Dietary Supplement
    Intervention Name(s)
    Omegaven
    Intervention Description
    After the diagnosis of PNALD is made, patients who are followed by the Neonatal Intensive Care Unit, NICU, will contact Dr. Honrubia and Dr. Swarup. Cases may also include referrals of patients with PNALD from other healthcare facilities or self-referrals. If the patient's parents or guardians agree to participate in the study, informed consent will be obtained. The history of present illness and past medical history will be reviewed with the guardian and pertinent demographic and medical information will be recorded on data collection forms. This form will be used to record all laboratory results, nutritional history, and descriptions of any liver biopsies performed.

    10. Eligibility

    Sex
    All
    Minimum Age & Unit of Time
    22 Weeks
    Maximum Age & Unit of Time
    40 Weeks
    Eligibility Criteria
    Inclusion Criteria: - 1. Inborn and transferred newborns born between the gestational age of 22-40 weeks who develop chronic liver failure, secondary to parenteral nutrition. 2. Patients will be PN dependent (unable to meet nutritional needs soley by enteral nutrition) and are expected to require PN for at least another 30 days 3. Patients must have diagnosis of parenteral nutrition associated liver disease (PNALD) as defined by serum direct bilirubin greater than 2mg/dL on 2 consecutive occasions 4. have had PNALD for at least four weeks prior to planned Omegaven initiation 5. Direct bilirubin > 2.0 mg/dl 6. Signed patient informed consent. 7. The patient must have utilized standard therapies to prevent the progression of his/her liver disease including surgical treatment, cyclic PN, avoiding overfeeding, reduction/removal of copper and manganese from PN, advancement of enteral feeding, and the use of ursodiol (i..e., Actigall®). Exclusion Criteria: - 2. Other causes of chronic liver disease (Hepatitis C, Cystic fibrosis, biliary atresia, and alpha 1 anti-trypsin deficiency,) 3. severe and/or unstable concomitant systemic disease such as complex congenital cardiac disease, renal failure, autoimmune disease, sepsis, inborn error of metabolism, genetic liver disease, 4. bleeding disorder; 5. biochemical disturbance with potential of worsening with proposed treatment, e.g. persistent hyperglycemia, hypertriglyceridemia, hypercalcemia. 6. The parent or guardian or child unwilling to provide consent or assent In rare instances, patients diagnosed with PNALD may later be found to have liver disease due to other causes in addition to the use of PN (i.e., inborn errors of metabolism, viral infections ). Such causes may not be known at the time of enrollment and will not preclude them from continuing in the study.

    12. IPD Sharing Statement

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