Trial Outcomes & Findings for Compassionate Use of Omegaven in Children (NCT NCT02370251)

NCT ID: NCT02370251

Last Updated: 2019-11-25

Results Overview

To determine if Omegaven results in the resolution of cholestasis (DB \<2 for 2 consecutive weeks)

• Recruitment status: COMPLETED
• Study phase: PHASE2/PHASE3
• Target enrollment: 63 participants
• Primary outcome timeframe: Within the first 3 months of sole Omegaven use
• Results posted on: 2019-11-25

Participant Flow

Participant milestones

Measure	Omegaven 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.
Overall Study STARTED	63
Overall Study COMPLETED	52
Overall Study NOT COMPLETED	11

Reasons for withdrawal

Measure	Omegaven 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.
Overall Study Received less than 8 days of Omegaven	7
Overall Study Death	4

Baseline Characteristics

Compassionate Use of Omegaven in Children

Baseline characteristics by cohort

Measure	Omegaven n=52 Participants 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.
Age, Customized Birth Gestational Age	30 weeks n=5 Participants
Sex: Female, Male Female	23 Participants n=5 Participants
Sex: Female, Male Male	29 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	8 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	44 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	2 Participants n=5 Participants
Race (NIH/OMB) Asian	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	6 Participants n=5 Participants
Race (NIH/OMB) White	43 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	52 participants n=5 Participants
Birth Weight (g)	1410 grams n=5 Participants

PRIMARY outcome

Timeframe: 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)

Outcome measures

Measure	Omegaven n=52 Participants 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.
Resolution of Cholestasis for Subjects Who Received Omegaven Cholestasis resolved within 3 months	40 Participants
Resolution of Cholestasis for Subjects Who Received Omegaven Cholestasis resolved after 3 months	10 Participants
Resolution of Cholestasis for Subjects Who Received Omegaven Cholestasis not resolved	2 Participants

SECONDARY outcome

Timeframe: 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

Outcome measures

Measure	Omegaven n=52 Participants 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.
Safety Issues for Infants Who Received Omegaven Baseline : INR >1.4	10 Participants
Safety Issues for Infants Who Received Omegaven End of Treatment : INR >1.4	7 Participants
Safety Issues for Infants Who Received Omegaven Baseline : Gamma-Glutamyl Transferase (GGT) >300	6 Participants
Safety Issues for Infants Who Received Omegaven End of Treatment : GGT >300	7 Participants
Safety Issues for Infants Who Received Omegaven Baseline : High-density Lipoprotein (HDL) <20	39 Participants
Safety Issues for Infants Who Received Omegaven End of Treatment : HDL <20	24 Participants

SECONDARY outcome

Timeframe: Within the first month of use

To determine if Omegaven can resolve essential fatty acid deficiency

Outcome measures

Measure	Omegaven n=52 Participants 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.
Essential Fatty Acid Deficiency in Infants Who Received Omegaven Did not have EFAD	14 Participants
Essential Fatty Acid Deficiency in Infants Who Received Omegaven EFAD resolved	27 Participants
Essential Fatty Acid Deficiency in Infants Who Received Omegaven EFAD not resolved	11 Participants

Adverse Events

Omegaven

Serious events: 9 serious events

Other events: 0 other events

Deaths: 4 deaths

Serious adverse events

Measure	Omegaven n=52 participants at risk 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.
Blood and lymphatic system disorders Elevated INR	7.7% 4/52 • 1 year
Hepatobiliary disorders Elevated GGT	9.6% 5/52 • 1 year

Other adverse events

Adverse event data not reported

Additional Information

Kimberly Ernst, MD

University of Oklahoma

Phone: 405-271-5215

Email: kimberly-ernst@ouhsc.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place