Parenteral Nutrition-related Hepatitis
Conditions
Brief summary
To provide parenteral fat emulsion prepared from fish oil (omega-3 fatty acid emulsion, Omegaven®) to patients with liver disease (PNALD).
Detailed description
This is a compassionate use protocol for patients with PNALD who have failed traditional treatment to receive Omegaven through an IND. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition.
Interventions
DRUGOmegaven
Expanded Access Therapy with Omegaven will be initiated at a starting dose of 0.5 gm/kg/day to be infused over 24 hrs. After two days of infusion the dose will be increased to 1gm/kg/day.
Sponsors
Children's Mercy Hospital Kansas City
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
This is a compassionate use protocol for patients with PNALD who have failed traditional treatment to receive Omegaven through an IND.
Eligibility
Sex/Gender
ALL
Age
No minimum to 17 Years
Healthy volunteers
No
Inclusion criteria
1. The patient will be PN dependent and unable to meet nutritional needs solely by enteral nutrition. 2. Patient will be \<18 years of age. 3. Direct bilirubin \> 2.0mg/dl 4. The patient must have failed standard therapies to prevent progression his/her liver disease.
Exclusion criteria
1. Other causes of chronic liver disease (Hepatitis C, Cystic fibrosis, biliary atresia, and alpha 1 anti-trypsin deficiency). 2. Patients who are allergic to eggs/shellfish 3. Patients who have severe hemorrhagic disorders.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Disease Progression as Measured by Serum Levels of Hepatic Enzymes | Up to 1 year | To evaluate whether established parenteral nutrition associated liver disease (PNALD) can be reversed or its progression halted by using a parenteral fat emulsion prepared from fish oil (omega-3 fatty acid emulsion, Omegaven®) as measured by normalization or decrease of serum levels of hepatic enzymes. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Overall Study Use of Omegaven for patients with parenteral nutrition associated liver disease.
Omegaven: Expanded Access Therapy with Omegaven will be initiated at a starting dose of 0.5 gm/kg/day to be infused over 24 hrs. After two days of infusion the dose will be increased to 1gm/kg/day. | 2 |
| Total | 2 |
Baseline characteristics
| Characteristic | Overall Study | — |
|---|---|---|
| Age, Continuous | 4.42 Years STANDARD_DEVIATION 3.58 | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Sex: Female, Male Female | 0 Participants | — |
| Sex: Female, Male Male | 2 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 2 |
| other Total, other adverse events | 0 / 2 |
| serious Total, serious adverse events | 0 / 2 |
Outcome results
Primary
Disease Progression as Measured by Serum Levels of Hepatic Enzymes
To evaluate whether established parenteral nutrition associated liver disease (PNALD) can be reversed or its progression halted by using a parenteral fat emulsion prepared from fish oil (omega-3 fatty acid emulsion, Omegaven®) as measured by normalization or decrease of serum levels of hepatic enzymes.
Time frame: Up to 1 year
Population: No results recorded, study was utilized to provide compassionate use of study medication to participants.