Cholestasis, Cholestasis of Parenteral Nutrition, Gastroschisis, Infant, Premature, Diseases, Intestinal Atresia, Short Bowel Syndrome, Total Parenteral Nutrition-induced Cholestasis
Conditions
Keywords
Fat Emulsions, Intravenous, Parenteral-Nutrition-Associated Liver Disease, Cholestasis, Omegaven, Fish Oil
Brief summary
This is a single-assignment study to evaluate whether Omegaven (IV fish oil) is effective at treating liver disease in children on long-term IV nutrition.
Detailed description
Children up to 18 years of age, on parenteral nutrition, with a direct bilirubin level of 2 mg/dL or greater will be eligible to receive Omegaven at a maximum dose of 1 g/kg/day. Up to 200 children will be eligible for enrollment. Direct bilirubin levels and other labs will be monitored as well as growth parameters.
Interventions
DRUGOmegaven
Once the direct bilirubin is 2 mg/dL or more x 2 weeks, Intralipid will be switched to Omegaven at 1 g/kg/day. The bilirubin level will be monitored to determine when resolution of cholestasis (DB \<2 mg/dL) occurs.
Sponsors
OU Medical Center
University of Oklahoma
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
Healthy volunteers
No
Inclusion criteria
* Live in or temporarily relocate to Oklahoma * Age less than 18 years, both sexes, all races * Have a direct bilirubin level of ≥2 mg/dL for two consecutive weeks after at least 14 days of parenteral nutrition * Received parenteral lipids at a maximum dose of 1.7 g/kg/day (24 g/kg over the two weeks prior) * Are not currently enrolled in another lipid emulsion study
Exclusion criteria
* Known food allergy to fish * Known metabolic disorder of lipid metabolism * Active coagulopathies (active bleeding or requiring blood product treatment in the prior 48 hours) * Medical condition likely to result in death in the next 30 days
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Resolution of Cholestasis for Subjects Who Received Omegaven | Within the first 3 months of sole Omegaven use | To determine if Omegaven results in the resolution of cholestasis (DB \<2 for 2 consecutive weeks) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Safety Issues for Infants Who Received Omegaven | Within the first year of use | To determine if Omegaven results in safety issues such as increased bleeding, essential fatty acid deficiency, elevated liver function tests, increased liver and/or intestinal transplant rates, or death |
| Essential Fatty Acid Deficiency in Infants Who Received Omegaven | Within the first month of use | To determine if Omegaven can resolve essential fatty acid deficiency |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Omegaven Children will receive Omegaven at a maximum of 1 g/kg/day upon enrollment in this arm.
Omegaven: Once the direct bilirubin is 2 mg/dL or more x 2 weeks, Intralipid will be switched to Omegaven at 1 g/kg/day. The bilirubin level will be monitored to determine when resolution of cholestasis (DB \<2 mg/dL) occurs. | 52 |
| Total | 52 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Death | 4 |
| Overall Study | Received less than 8 days of Omegaven | 7 |
Baseline characteristics
| Characteristic | Omegaven |
|---|---|
| Age, Customized Birth Gestational Age | 30 weeks |
| Birth Weight (g) | 1410 grams |
| Ethnicity (NIH/OMB) Hispanic or Latino | 8 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 44 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 2 Participants |
| Race (NIH/OMB) Asian | 1 Participants |
| Race (NIH/OMB) Black or African American | 6 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 43 Participants |
| Region of Enrollment United States | 52 participants |
| Sex: Female, Male Female | 23 Participants |
| Sex: Female, Male Male | 29 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 4 / 52 |
| other Total, other adverse events | 0 / 52 |
| serious Total, serious adverse events | 9 / 52 |
Outcome results
Primary
Resolution of Cholestasis for Subjects Who Received Omegaven
To determine if Omegaven results in the resolution of cholestasis (DB \<2 for 2 consecutive weeks)
Time frame: Within the first 3 months of sole Omegaven use
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Omegaven | Resolution of Cholestasis for Subjects Who Received Omegaven | Cholestasis resolved within 3 months | 40 Participants |
| Omegaven | Resolution of Cholestasis for Subjects Who Received Omegaven | Cholestasis resolved after 3 months | 10 Participants |
| Omegaven | Resolution of Cholestasis for Subjects Who Received Omegaven | Cholestasis not resolved | 2 Participants |
Secondary
Essential Fatty Acid Deficiency in Infants Who Received Omegaven
To determine if Omegaven can resolve essential fatty acid deficiency
Time frame: Within the first month of use
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Omegaven | Essential Fatty Acid Deficiency in Infants Who Received Omegaven | Did not have EFAD | 14 Participants |
| Omegaven | Essential Fatty Acid Deficiency in Infants Who Received Omegaven | EFAD resolved | 27 Participants |
| Omegaven | Essential Fatty Acid Deficiency in Infants Who Received Omegaven | EFAD not resolved | 11 Participants |
Secondary
Safety Issues for Infants Who Received Omegaven
To determine if Omegaven results in safety issues such as increased bleeding, essential fatty acid deficiency, elevated liver function tests, increased liver and/or intestinal transplant rates, or death
Time frame: Within the first year of use
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Omegaven | Safety Issues for Infants Who Received Omegaven | Baseline : INR >1.4 | 10 Participants |
| Omegaven | Safety Issues for Infants Who Received Omegaven | End of Treatment : INR >1.4 | 7 Participants |
| Omegaven | Safety Issues for Infants Who Received Omegaven | Baseline : Gamma-Glutamyl Transferase (GGT) >300 | 6 Participants |
| Omegaven | Safety Issues for Infants Who Received Omegaven | End of Treatment : GGT >300 | 7 Participants |
| Omegaven | Safety Issues for Infants Who Received Omegaven | Baseline : High-density Lipoprotein (HDL) <20 | 39 Participants |
| Omegaven | Safety Issues for Infants Who Received Omegaven | End of Treatment : HDL <20 | 24 Participants |