Use of Omegaven for Treatment With Parenteral Nutrition Associated Liver Disease
Study Details
Study Description
Brief Summary
To provide parenteral fat emulsion prepared from fish oil (omega-3 fatty acid emulsion, Omegaven®) to patients with liver disease (PNALD).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
| Phase 2/Phase 3 |
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.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Overall Study Use of Omegaven for patients with parenteral nutrition associated liver disease. |
Drug: 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.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- 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.
Eligibility Criteria
Criteria
Inclusion Criteria:
-
The patient will be PN dependent and unable to meet nutritional needs solely by enteral nutrition.
-
Patient will be <18 years of age.
-
Direct bilirubin > 2.0mg/dl
-
The patient must have failed standard therapies to prevent progression his/her liver disease.
Exclusion Criteria:
-
Other causes of chronic liver disease (Hepatitis C, Cystic fibrosis, biliary atresia, and alpha 1 anti-trypsin deficiency).
-
Patients who are allergic to eggs/shellfish
-
Patients who have severe hemorrhagic disorders.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | Children's Mercy Hospitals and Clinics | Kansas City | Missouri | United States | 64108 |
Sponsors and Collaborators
- Children's Mercy Hospital Kansas City
Investigators
- Principal Investigator: Joel D Lim, MD, Children's Mercy Hospital Kansas City
Study Documents (Full-Text)
More Information
Publications
None provided.- 12010063
Study Results
Participant Flow
| Recruitment Details | |
|---|---|
| Pre-assignment Detail |
| Arm/Group Title | Overall Study |
|---|---|
| Arm/Group Description | 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. |
| Period Title: Overall Study | |
| STARTED | 2 |
| COMPLETED | 2 |
| NOT COMPLETED | 0 |
Baseline Characteristics
| Arm/Group Title | Overall Study |
|---|---|
| Arm/Group Description | 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. |
| Overall Participants | 2 |
| Age (Years) [Mean (Standard Deviation) ] | |
| Mean (Standard Deviation) [Years] |
4.42
(3.58)
|
| Sex: Female, Male (Count of Participants) | |
| Female |
0
0%
|
| Male |
2
100%
|
| Race and Ethnicity Not Collected (Count of Participants) | |
Outcome Measures
| Title | Disease Progression as Measured by Serum Levels of Hepatic Enzymes |
|---|---|
| Description | 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 |
Outcome Measure Data
| Analysis Population Description |
|---|
| No results recorded, study was utilized to provide compassionate use of study medication to participants. |
| Arm/Group Title | Overall Study |
|---|---|
| Arm/Group Description | 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. |
| Measure Participants | 0 |
Adverse Events
| Time Frame | 1 year | |
|---|---|---|
| Adverse Event Reporting Description | ||
| Arm/Group Title | Overall Study | |
| Arm/Group Description | 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. | |
All Cause Mortality |
||
| Overall Study | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/2 (0%) | |
Serious Adverse Events |
||
| Overall Study | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/2 (0%) | |
Other (Not Including Serious) Adverse Events |
||
| Overall Study | ||
| Affected / at Risk (%) | # Events | |
| Total | 0/2 (0%) | |
Limitations/Caveats
More Information
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
| Name/Title | Joel Lim, MD - Principal Investigator |
|---|---|
| Organization | Children's Mercy Hospital |
| Phone | (816)234-3000 |
| jlim@cmh.edu |
- 12010063