Clinical trial NCT03662282: Omegaven as Alternative Parenteral Fat Nutrition
Official title: Omegaven as Alternative Parenteral Fat Nutrition
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cholestasis
Cholestasis of Parenteral Nutrition
Keywords: Omegaven
parenteral nutrition
Liver disease
Cholestasis
Lead sponsor: University of South Florida
Sponsor class: OTHER
First posted: 2018-09-07
Enrollment: 2
Sex: ALL
Minimum age (years): 14
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: To provide Omegaven® as a compassionate treatment for critically ill infants with parenteral nutrition associated cholestasis

Detailed description: An intermediate population, Investigational New Drug study, to provide an alternate intravenous fat nutrition, Omegaven®, to help improve liver function while providing adequate nutrition for critically ill infants with parenteral nutrition associated cholestasis

Eligibility criteria: Inclusion Criteria:

* Greater than 14 days old
* Has parenteral nutrition associated cholestasis defined as at least 2 consecutive direct bilirubin \>2 mg/dL with anatomical or functional short gut (OR \>4 mg/dL if intact intestine) obtained at least 1 week apart with a ratio of direct: total bilirubin \> 0.4
* Patient is parenteral nutrition dependent (unable to meet nutritional needs solely by enteral nutrition) and are expected to require parenteral nutrition for at least 3 more weeks
* Patient has not responded to other therapeutic approaches for parenteral nutrition-associated liver disease such as : cycling of parenteral nutrition, avoiding overfeeding, reduction/removal of copper and manganese from parenteral nutrition, advancement of enteral feeding, and use of Ursodiol
* Patient has been on at least 2 weeks of SMOFLIPIDs with no improvement or worsening of direct bilirubin levels
* Signed patient informed consent
* The patient is expected to have a reasonable possibility of survival
* No other known etiology of cholestasis other than parenteral nutrition-associated liver disease at the time of Omegaven® initiation

Exclusion Criteria:

* Known causes of cholestasis other than parenteral nutrition-associated liver disease including but not limited to Hepatitis C, Cystic fibrosis, biliary atresia, and alpha 1 antitrypsin deficiency are present, prior to Omegaven® initiation
* Known fish or egg allergy
* Any of the contraindications to use of Omegaven®:

  1. Active new infection at the time of initiation of Omegaven®
  2. Hemodynamic instability
  3. Recent use of medications with associated risk of bleeding, including NSAIDs
  4. Active coagulopathy or bleeding
  5. Platelet counts persistently under 30,000 despite transfusions
  6. Unstable hyperglycemia
  7. Impaired lipid metabolism (triglycerides \>1000 mg/dL) while on 1 g/kg/day or less of Intralipid
  8. History of severe hemorrhagic disorders (ie. hemophilia, Von Willebrand disease, etc.)
  9. Unstable diabetes mellitus
  10. Collapse and shock
  11. Stroke/ Embolism
  12. Cardiac infarction within the last 3 months
  13. Undefined coma status

Primary outcomes:
measure: Number of Participant With Decrease in Direct Bilirubin Level
timeFrame: First month of treatment
Secondary outcomes:
measure: Number of Participant With Resolution of Direct Hyperbilirubinemia
timeFrame: 2 months
measure: Number of Participant With Preservation of Length
timeFrame: Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment
measure: Number of Participant With Preservation of Head Circumference
timeFrame: Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment
measure: Number of Participant With Preservation of Normal Weight Gain
timeFrame: Weekly measurement during treatment. Evaluation every 30 days from treatment initiation until discontinuation of treatment, patient hospital discharge, whichever comes first, or up to 6 months following initiation of treatment
Other outcomes:
Locations:
city: Tampa
country: United States
facility: USF Health
state: Florida
Study officials:
affiliation: University of South Florida
name: Thao Ho, DO
role: PRINCIPAL_INVESTIGATOR
References: