Trial Outcomes & Findings for Low Dose Fat for the Prevention of Liver Disease in Babies With Gastrointestinal Disorders (NCT NCT01373918)

NCT ID: NCT01373918

Last Updated: 2016-06-16

Results Overview

Cholestasis will be defined by a direct bilirubin \> 2 mg/dL

• Recruitment status: TERMINATED
• Study phase: PHASE4
• Target enrollment: 41 participants
• Primary outcome timeframe: prior to 100 days of life, hospital discharge, or death whichever comes first
• Results posted on: 2016-06-16

Participant Flow

41 subjects were enrolled in the study. Only subjects who required > 14 days of parenteral nutrition and who had an abdominal surgery were included in the final analysis.

Participant milestones

Measure	Low Dose Intravenous Fat Emulsion Subjects in this arm will receive approximately 1 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 1 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.	Standard Dose Intravenous Fat Emulsion Subjects in this arm will receive approximately 3 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 3 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.
Overall Study STARTED	20	16
Overall Study COMPLETED	20	15
Overall Study NOT COMPLETED	0	1

Reasons for withdrawal

Measure	Low Dose Intravenous Fat Emulsion Subjects in this arm will receive approximately 1 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 1 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.	Standard Dose Intravenous Fat Emulsion Subjects in this arm will receive approximately 3 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 3 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.
Overall Study Physician Decision	0	1

Baseline Characteristics

Low Dose Fat for the Prevention of Liver Disease in Babies With Gastrointestinal Disorders

Baseline characteristics by cohort

Measure	Low Dose Intravenous Fat Emulsion n=20 Participants Subjects in this arm will receive approximately 1 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 1 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.	Standard Dose Intravenous Fat Emulsion n=16 Participants Subjects in this arm will receive approximately 3 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 3 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.	Total n=36 Participants Total of all reporting groups
Age, Continuous	1.3 days STANDARD_DEVIATION 1.4 • n=5 Participants	1.3 days STANDARD_DEVIATION 1.1 • n=7 Participants	1.3 days STANDARD_DEVIATION 1.3 • n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants	6 Participants n=7 Participants	18 Participants n=5 Participants
Sex: Female, Male Male	8 Participants n=5 Participants	10 Participants n=7 Participants	18 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	7 Participants n=5 Participants	11 Participants n=7 Participants	18 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	13 Participants n=5 Participants	5 Participants n=7 Participants	18 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) White	17 Participants n=5 Participants	14 Participants n=7 Participants	31 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants	16 participants n=7 Participants	36 participants n=5 Participants

PRIMARY outcome

Timeframe: prior to 100 days of life, hospital discharge, or death whichever comes first

Cholestasis will be defined by a direct bilirubin > 2 mg/dL

Outcome measures

Measure	Low Dose Intravenous Fat Emulsion n=20 Participants Subjects in this arm will receive approximately 1 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 1 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.	Standard Dose Intravenous Fat Emulsion n=16 Participants Subjects in this arm will receive approximately 3 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 3 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.
Presence of Cholestasis Presence of Cholestasis-Yes	6 participants	6 participants
Presence of Cholestasis Presence of Cholestasis-No	14 participants	10 participants

SECONDARY outcome

Timeframe: at the end of the hospital stay which is expected to be an average of 5 weeks

death

Outcome measures

Measure	Low Dose Intravenous Fat Emulsion n=20 Participants Subjects in this arm will receive approximately 1 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 1 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.	Standard Dose Intravenous Fat Emulsion n=16 Participants Subjects in this arm will receive approximately 3 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 3 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.
Mortality Rate	0 participants	0 participants

SECONDARY outcome

Timeframe: 28 days of age

Growth will be assessed by growth velocity at 28 days of age

Outcome measures

Measure	Low Dose Intravenous Fat Emulsion n=20 Participants Subjects in this arm will receive approximately 1 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 1 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.	Standard Dose Intravenous Fat Emulsion n=16 Participants Subjects in this arm will receive approximately 3 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 3 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.
Anthropometric Measurements	18 g/d Standard Deviation 8	24 g/d Standard Deviation 16

SECONDARY outcome

Timeframe: approximately 5 weeks

Growth will be assessed by weight at the time of hospital discharge (approximately 5 weeks)

Outcome measures

Measure	Low Dose Intravenous Fat Emulsion n=20 Participants Subjects in this arm will receive approximately 1 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 1 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.	Standard Dose Intravenous Fat Emulsion n=16 Participants Subjects in this arm will receive approximately 3 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 3 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.
Anthropometric Measurements	3.4 g Standard Deviation 0.9	3.5 g Standard Deviation 0.7

Adverse Events

Low Dose Intravenous Fat Emulsion

Serious events: 2 serious events

Other events: 0 other events

Deaths: 0 deaths

Standard Dose Intravenous Fat Emulsion

Serious events: 1 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Measure	Low Dose Intravenous Fat Emulsion n=20 participants at risk Subjects in this arm will receive approximately 1 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 1 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.	Standard Dose Intravenous Fat Emulsion n=16 participants at risk Subjects in this arm will receive approximately 3 g/kg/d IV of intravenous soybean oil (Intralipid). Intralipid: The subject will receive 3 g/kg/d of the standard intravenous fat emulsion while receiving Parenteral Nutrition until discharge from the hospital, death or 100 days of life, whichever comes first.
Metabolism and nutrition disorders hyperglycemia	10.0% 2/20 • Number of events 2 • Til hospital discharge, which is an average of approximately 5 weeks	0.00% 0/16 • Til hospital discharge, which is an average of approximately 5 weeks
Metabolism and nutrition disorders hypertriglyceridemia	0.00% 0/20 • Til hospital discharge, which is an average of approximately 5 weeks	6.2% 1/16 • Number of events 2 • Til hospital discharge, which is an average of approximately 5 weeks

Other adverse events

Adverse event data not reported

Additional Information

Dr. Kara L. Calkins

University of California, Los Angeles

Phone: 310 825-9330

Email: KCalkins@mednet.ucla.edu

Results disclosure agreements

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