Compassionate Use of Omegaven in Children

• Sponsor: University of Oklahoma (Other)
• Overall Status: Completed
• CT.gov ID: NCT02370251
• Collaborator: OU Medical Center (Other)
• Enrollment: 63
• Location: 1
• Arm: 1
• Actual Duration (Months): 51.9
• Patients Per Site Per Month: 1.2

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Total Parenteral Nutrition-induced Cholestasis; Cholestasis; Short Bowel Syndrome; Gastroschisis; Intestinal Atresia; Infant, Premature, Diseases; Cholestasis of Parenteral Nutrition	Drug: Omegaven	Phase 2/Phase 3

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.

Study Design

• Study Type: Interventional
• Actual Enrollment: 63 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Fish Oil Lipid Emulsion for the Treatment of Parenteral-Nutrition-Associated Liver Disease in Infants
• Actual Study Start Date: Jun 1, 2015
• Actual Primary Completion Date: Sep 27, 2018
• Actual Study Completion Date: Sep 27, 2019

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Omegaven Children will receive Omegaven at a maximum of 1 g/kg/day upon enrollment in this arm.	Drug: 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.; Other Names: Fish oil-based lipid Omega-3-fatty acid lipid emulsion

Outcome Measures

Primary Outcome Measures

1. 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 Outcome Measures

1. 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

2. 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

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A to 18 Years
• Sexes Eligible for Study: All
• Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	OU Children's Hospital	Oklahoma City	Oklahoma	United States	73104

Sponsors and Collaborators

• University of Oklahoma
• OU Medical Center

Investigators

• Principal Investigator: Kimberly D Ernst, MD, MSMI, The University of Oklahoma, Department of Pediatrics

Study Documents (Full-Text)

• Study Protocol and Statistical Analysis Plan - Feb 19, 2018

More Information

Publications

None provided.

• Responsible Party: University of Oklahoma
• ClinicalTrials.gov Identifier: NCT02370251
• Other Study ID Numbers: 5451
• First Posted: Feb 24, 2015
• Last Update Posted: Nov 25, 2019
• Last Verified: Nov 1, 2019

• Keywords provided by University of Oklahoma: Fat Emulsions, Intravenous; Parenteral-Nutrition-Associated Liver Disease; Cholestasis; Omegaven; Fish Oil
• Additional relevant MeSH terms: Gastroschisis; Cholestasis; Short Bowel Syndrome; Intestinal Atresia; Infant, Premature, Diseases

Study Results

Participant Flow

Arm/Group Title	Omegaven
Arm/Group Description	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.
Period Title: Overall Study
STARTED	63
COMPLETED	52
NOT COMPLETED	11

Baseline Characteristics

Arm/Group Title	Omegaven
Arm/Group Description	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.
Overall Participants	52
Age, Customized (weeks) [Mean (Inter-Quartile Range) ]
Birth Gestational Age	30
Sex: Female, Male (Count of Participants)
Female	23 44.2%
Male	29 55.8%
Ethnicity (NIH/OMB) (Count of Participants)
Hispanic or Latino	8 15.4%
Not Hispanic or Latino	44 84.6%
Unknown or Not Reported	0 0%
Race (NIH/OMB) (Count of Participants)
American Indian or Alaska Native	2 3.8%
Asian	1 1.9%
Native Hawaiian or Other Pacific Islander	0 0%
Black or African American	6 11.5%
White	43 82.7%
More than one race	0 0%
Unknown or Not Reported	0 0%
Region of Enrollment (participants) [Number]
United States	52 100%
Birth Weight (g) (grams) [Mean (Inter-Quartile Range) ]
Mean (Inter-Quartile Range) [grams]	1410

Outcome Measures

1. Primary Outcome

Title	Resolution of Cholestasis for Subjects Who Received Omegaven
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Omegaven
Arm/Group Description	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.
Measure Participants	52
Cholestasis resolved within 3 months	40 76.9%
Cholestasis resolved after 3 months	10 19.2%
Cholestasis not resolved	2 3.8%

2. Secondary Outcome

Title	Safety Issues for Infants Who Received Omegaven
Description	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

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Omegaven
Arm/Group Description	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.
Measure Participants	52
Baseline : INR >1.4	10 19.2%
End of Treatment : INR >1.4	7 13.5%
Baseline : Gamma-Glutamyl Transferase (GGT) >300	6 11.5%
End of Treatment : GGT >300	7 13.5%
Baseline : High-density Lipoprotein (HDL) <20	39 75%
End of Treatment : HDL <20	24 46.2%

3. Secondary Outcome

Title	Essential Fatty Acid Deficiency in Infants Who Received Omegaven
Description	To determine if Omegaven can resolve essential fatty acid deficiency
Time Frame	Within the first month of use

Outcome Measure Data

Analysis Population Description

[Not Specified]

Arm/Group Title	Omegaven
Arm/Group Description	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.
Measure Participants	52
Did not have EFAD	14 26.9%
EFAD resolved	27 51.9%
EFAD not resolved	11 21.2%

Adverse Events

Time Frame	1 year
Arm/Group Title	Omegaven
Arm/Group Description	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.
All Cause Mortality
	Omegaven
	Affected / at Risk (%)	# Events
Total	4/52 (7.7%)
Serious Adverse Events
	Omegaven
	Affected / at Risk (%)	# Events
Total	9/52 (17.3%)
Blood and lymphatic system disorders
Elevated INR	4/52 (7.7%)
Hepatobiliary disorders
Elevated GGT	5/52 (9.6%)
Other (Not Including Serious) Adverse Events
	Omegaven
	Affected / at Risk (%)	# Events
Total	0/52 (0%)

Limitations/Caveats

This was a compassionate use study, using Omegaven as a "last resort" for infants with a direct bilirubin of at least 2 mg/dL at the time of initiation, so these infants were already at higher risk of death and hematological/hepatobiliary morbidity.

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.