Trial Outcomes & Findings for Standard Lipid Therapy vs IVFE Minimization for Prevention of PNALD (NCT NCT02357576)
NCT ID: NCT02357576
Last Updated: 2020-12-17
Results Overview
The rate of rise (change over time) of direct bilirubin was compared between the two groups at different time points.
COMPLETED
PHASE3
22 participants
12 weeks
2020-12-17
Participant Flow
Patients who were enrolled were randomized shortly after enrollment. 1 patient was removed immediately after enrollment, before intervention, due to a screen fail.
Participant milestones
| Measure |
Reduced Lipid
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Treatment Period
STARTED
|
12
|
9
|
|
Treatment Period
COMPLETED
|
12
|
7
|
|
Treatment Period
NOT COMPLETED
|
0
|
2
|
|
Neurodevelopmental Outcome Follow Up
STARTED
|
11
|
7
|
|
Neurodevelopmental Outcome Follow Up
COMPLETED
|
6
|
2
|
|
Neurodevelopmental Outcome Follow Up
NOT COMPLETED
|
5
|
5
|
Reasons for withdrawal
| Measure |
Reduced Lipid
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Treatment Period
Withdrawal by Subject
|
0
|
1
|
|
Treatment Period
Physician Decision
|
0
|
1
|
|
Neurodevelopmental Outcome Follow Up
Scheduling error
|
1
|
1
|
|
Neurodevelopmental Outcome Follow Up
Became ineligible during follow-up
|
1
|
0
|
|
Neurodevelopmental Outcome Follow Up
Lost to Follow-up
|
1
|
4
|
|
Neurodevelopmental Outcome Follow Up
Withdrawal by Subject
|
2
|
0
|
Baseline Characteristics
Standard Lipid Therapy vs IVFE Minimization for Prevention of PNALD
Baseline characteristics by cohort
| Measure |
Reduced Lipid
n=12 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=9 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Total
n=21 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Race/Ethnicity, Customized
Asian
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Age, Continuous
|
2 days
n=5 Participants
|
2 days
n=7 Participants
|
2 days
n=5 Participants
|
|
Sex: Female, Male
Female
|
8 Participants
n=5 Participants
|
3 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
0 participants
n=5 Participants
|
0 participants
n=7 Participants
|
0 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
3 participants
n=5 Participants
|
0 participants
n=7 Participants
|
3 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
1 participants
n=5 Participants
|
1 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
White
|
8 participants
n=5 Participants
|
5 participants
n=7 Participants
|
13 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 participants
n=5 Participants
|
1 participants
n=7 Participants
|
1 participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Unknown
|
0 participants
n=5 Participants
|
2 participants
n=7 Participants
|
2 participants
n=5 Participants
|
|
Diagnosis
Gastroschisis
|
12 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
20 Participants
n=5 Participants
|
|
Diagnosis
Intestinal atresia
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 weeksPopulation: Analyzable data was not available for one participant in the Reduced Lipid Arm
The rate of rise (change over time) of direct bilirubin was compared between the two groups at different time points.
Outcome measures
| Measure |
Reduced Lipid
n=11 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=9 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Rate of Rise of Direct Bilirubin as a Function of Time
1 Day from Baseline
|
1.033001353 mg/dL/day
Interval 1.011427197 to 1.055035694
|
1.05457485 mg/dL/day
Interval 1.029533513 to 1.080225375
|
|
Rate of Rise of Direct Bilirubin as a Function of Time
7 Days from Baseline
|
1.025504411 mg/dL/day
Interval 1.005132124 to 1.046289609
|
1.049318179 mg/dL/day
Interval 1.025241711 to 1.07396016
|
|
Rate of Rise of Direct Bilirubin as a Function of Time
28 Days from Baseline
|
0.999691048 mg/dL/day
Interval 0.981391909 to 1.018331496
|
1.031125269 mg/dL/day
Interval 1.008439715 to 1.05432115
|
|
Rate of Rise of Direct Bilirubin as a Function of Time
56 Days from Baseline
|
0.96628119 mg/dL/day
Interval 0.945472667 to 0.987547779
|
1.007357736 mg/dL/day
Interval 0.981844238 to 1.033534209
|
|
Rate of Rise of Direct Bilirubin as a Function of Time
84 Days from Baseline
|
0.933987896 mg/dL/day
Interval 0.907126199 to 0.961645019
|
0.984138047 mg/dL/day
Interval 0.952398442 to 1.016935507
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: Analyzable data was not available for one participant in the Reduced Lipid Arm
The number of participants who had a direct bilirubin ≥2 mg/dL were compared between the standard and reduced lipid groups. Bilirubin data was collected from baseline until 7 days after PN has been discontinued, but not to exceed a total of 12 weeks.
Outcome measures
| Measure |
Reduced Lipid
n=11 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=9 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Prevalence of Parenteral Nutrition-associated Cholestasis (PNAC) (Direct Bilirubin ≥2 mg/dL)
|
2 Participants
|
3 Participants
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: Analyzable data was not available for one participant in the Reduced Lipid Arm
The number of participants with severe PNAC defined as a direct bilirubin ≥4 mg/dL were compared between the standard and reduced lipid groups. Bilirubin data was collected from baseline until 7 days after PN has been discontinued, but not to exceed a total of 12 weeks.
Outcome measures
| Measure |
Reduced Lipid
n=11 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=9 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Prevalence of Severe Parenteral Nutrition-associated Cholestasis (PNAC) (Direct Bilirubin ≥4 mg/dL in Subjects on Parenteral Nutrition for at Least 2 Weeks)
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: Only the subjects that developed PNAC were analyzed
The time to development was compared between the standard and reduced lipid groups.
Outcome measures
| Measure |
Reduced Lipid
n=2 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=3 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
The Time to Development of PNAC
|
19.50 days
Interval 14.0 to 25.0
|
39.00 days
Interval 29.0 to 42.0
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: Subjects who developed severe PNAC as defined by a direct bilirubin value of 4 mg/dL or greater
The time to development from randomization was compared between the standard and reduced lipid groups.
Outcome measures
| Measure |
Reduced Lipid
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=1 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
The Time to Development of Severe PNAC
|
—
|
42 Days
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: Subjects with total bilirubin values collected after 1 week
The peak (highest) total bilirubin collected from each subject from after week 1 to end of treatment. This was compared between the standard and reduced lipid groups.
Outcome measures
| Measure |
Reduced Lipid
n=12 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=9 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Peak Total Bilirubin Level
|
1.7 mg/dL
Interval 0.2 to 9.9
|
2.9 mg/dL
Interval 0.7 to 9.9
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: Subjects with direct bilirubin values collected after 1 week
The peak (highest) direct bilirubin collected from each subject from after week 1 to end of treatment. This was compared between the standard and reduced lipid groups.
Outcome measures
| Measure |
Reduced Lipid
n=11 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=8 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Peak Direct Bilirubin Level
|
0.8 mg/dL
Interval 0.3 to 2.5
|
1.7 mg/dL
Interval 0.6 to 4.0
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: All patients in the reduced lipid group were analyzed. The standard lipid group had essential fatty acid profile drawn for one patient as not all patients in the standard group required the essential fatty acid profile to be drawn.
The number of participants who experienced EFAD was compared between the standard and reduced lipid groups.
Outcome measures
| Measure |
Reduced Lipid
n=12 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=1 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
The Prevalence of Essential Fatty Acid Deficiency (EFAD)
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: All subjects aside from the screen fail. Z-scores were recorded while subjects were "on treatment" which meant that they were recorded until 7 days after parenteral nutrition (PN) had been discontinued, but not to exceed 12 weeks for subjects still on PN. This is why the participants analyzed each week is a different number and decreasing with time
Z-scores were compared between subjects in the two treatment groups by week. Z-scores are the number of standard deviations above (positive value) or below (negative value) the median on the FENTON and WHO growth charts. Fenton scores were used for infants born \<37 weeks gestation. WHO scores were used for infants born at ≥37 weeks gestation.
Outcome measures
| Measure |
Reduced Lipid
n=12 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=9 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Birth
|
-1.36 z-score
Interval -3.14 to 0.47
|
-0.93 z-score
Interval -1.71 to 1.06
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 1
|
-2.28 z-score
Interval -3.46 to -0.3
|
-1.85 z-score
Interval -2.26 to 0.65
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 2
|
-2.32 z-score
Interval -3.49 to -0.6
|
-1.3 z-score
Interval -2.38 to -0.03
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 3
|
-2.33 z-score
Interval -3.73 to -0.59
|
-1.485 z-score
Interval -2.47 to 0.44
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 4
|
-2.575 z-score
Interval -3.61 to -0.84
|
-1.59 z-score
Interval -2.01 to -1.19
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 5
|
-2.42 z-score
Interval -3.52 to -0.44
|
-1.78 z-score
Interval -2.34 to -1.04
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 6
|
-2.54 z-score
Interval -3.81 to -0.55
|
-1.88 z-score
Interval -2.49 to -0.89
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 7
|
-2.37 z-score
Interval -3.48 to -0.28
|
-1.62 z-score
Interval -2.53 to -0.71
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 8
|
-1.86 z-score
Interval -2.66 to -0.55
|
-1.745 z-score
Interval -2.68 to -0.81
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 9
|
-2.25 z-score
Interval -2.38 to -0.41
|
-1.745 z-score
Interval -2.46 to -1.03
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 10
|
-1.415 z-score
Interval -2.44 to -0.39
|
-1.785 z-score
Interval -2.55 to -1.02
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 11
|
-1.355 z-score
Interval -2.31 to -0.4
|
-1.885 z-score
Interval -2.41 to -1.36
|
|
Adequacy of Growth as Evaluated by Z-scores for Weight
Study Week 12
|
-1.33 z-score
Interval -2.36 to -0.3
|
-1.885 z-score
Interval -2.56 to -1.21
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: All subjects aside from the screen fail. Z-scores were recorded while subjects were "on treatment" which meant that they were recorded until 7 days after parenteral nutrition (PN) had been discontinued, but not to exceed 12 weeks for subjects still on PN. This is why the participants analyzed each week is a different number and decreasing with time
Z-scores were compared between subjects in the two treatment groups. Z-scores are the number of standard deviations above (positive value) or below (negative value) the median on the FENTON and WHO growth charts. Fenton scores were used for infants born \<37 weeks gestation. WHO scores were used for infants born at ≥37 weeks gestation.
Outcome measures
| Measure |
Reduced Lipid
n=12 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=9 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 9
|
-1.42 z-score
Interval -2.1 to -0.45
|
-2.865 z-score
Interval -3.37 to -2.36
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 10
|
-1.58 z-score
Interval -2.47 to -0.69
|
-2.62 z-score
Interval -3.3 to -1.94
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 11
|
-1.94 z-score
Interval -2.83 to -1.05
|
-2.81 z-score
Interval -3.59 to -2.03
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 12
|
-0.935 z-score
Interval -1.7 to -0.17
|
-2.775 z-score
Interval -3.19 to -2.36
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Birth
|
-1.06 z-score
Interval -3.84 to 0.29
|
-0.87 z-score
Interval -2.62 to 1.12
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 1
|
-1.365 z-score
Interval -4.41 to 0.0
|
-1.22 z-score
Interval -3.2 to 0.96
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 2
|
-1.53 z-score
Interval -4.22 to -0.58
|
-1.55 z-score
Interval -3.73 to 0.21
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 3
|
-2.19 z-score
Interval -4.77 to -0.65
|
-1.775 z-score
Interval -2.57 to 0.56
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 4
|
-2.15 z-score
Interval -4.6 to -0.56
|
-1.91 z-score
Interval -2.72 to 0.06
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 5
|
-3.21 z-score
Interval -4.57 to -0.28
|
-2.11 z-score
Interval -3.25 to -1.48
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 6
|
-1.81 z-score
Interval -4.52 to -0.13
|
-2.37 z-score
Interval -3.25 to -1.14
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 7
|
-1.56 z-score
Interval -3.37 to -0.98
|
-2.39 z-score
Interval -3.47 to -1.31
|
|
Adequacy of Growth as Evaluated by Z-scores for Height
Study Week 8
|
-0.98 z-score
Interval -1.71 to 2.93
|
-2.51 z-score
Interval -3.29 to -1.73
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: All subjects aside from the screen fail. Z-scores were recorded while subjects were "on treatment" which meant that they were recorded until 7 days after parenteral nutrition (PN) had been discontinued, but not to exceed 12 weeks for subjects still on PN. This is why the participants analyzed each week is a different number and decreasing with time
Z-scores were compared between subjects in the two treatment groups. Z-scores are the number of standard deviations above (positive value) or below (negative value) the median on the FENTON and WHO growth charts. Fenton scores were used for infants born \<37 weeks gestation. WHO scores were used for infants born at ≥37 weeks gestation.
Outcome measures
| Measure |
Reduced Lipid
n=12 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=9 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Birth
|
-0.99 z-score
Interval -4.12 to 0.82
|
-0.94 z-score
Interval -1.65 to 0.95
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 1
|
-1.18 z-score
Interval -3.8 to 0.25
|
-1.3 z-score
Interval -1.83 to 0.5
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 2
|
-1.36 z-score
Interval -3.91 to 0.04
|
-0.82 z-score
Interval -2.31 to -0.02
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 3
|
-1.67 z-score
Interval -3.66 to -0.03
|
-1 z-score
Interval -1.58 to 0.17
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 4
|
-1.745 z-score
Interval -2.74 to -0.81
|
-1.1 z-score
Interval -1.45 to -0.79
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 5
|
-1.225 z-score
Interval -2.81 to 0.15
|
-1.38 z-score
Interval -1.41 to -1.32
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 6
|
-1.34 z-score
Interval -2.77 to 0.23
|
-1.56 z-score
Interval -1.74 to -1.18
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 7
|
-0.79 z-score
Interval -1.6 to -0.12
|
-1.095 z-score
Interval -1.25 to -0.94
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 8
|
-0.845 z-score
Interval -1.17 to -0.08
|
-1.455 z-score
Interval -1.61 to -1.3
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 9
|
-1.44 z-score
Interval -1.49 to -0.24
|
-1.39 z-score
Interval -1.51 to -1.27
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 10
|
-0.84 z-score
Interval -1.75 to 0.07
|
-1.255 z-score
Interval -1.35 to -1.16
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 11
|
-0.935 z-score
Interval -2.05 to 0.18
|
-1.19 z-score
Interval -1.19 to -1.19
|
|
Adequacy of Growth as Evaluated by Z-scores for Head Circumference
Study Week 12
|
-0.63 z-score
Interval -1.15 to -0.11
|
-1.31 z-score
Interval -1.88 to -0.74
|
SECONDARY outcome
Timeframe: 12 weeksPopulation: Subjects who experienced an episode of suspected sepsis, catheter-related blood stream infection, or NEC
The number of episodes were compared between the standard and reduced lipid groups of suspected sepsis episodes, NEC, or catheter-related blood stream infections.
Outcome measures
| Measure |
Reduced Lipid
n=1 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=2 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Adverse Events, as Defined by Any Episode of Sepsis and Catheter-related Blood Stream Infections
Participant 2
|
—
|
3 episodes
|
|
Adverse Events, as Defined by Any Episode of Sepsis and Catheter-related Blood Stream Infections
Participant 3
|
1 episodes
|
—
|
|
Adverse Events, as Defined by Any Episode of Sepsis and Catheter-related Blood Stream Infections
Participant 1
|
—
|
2 episodes
|
SECONDARY outcome
Timeframe: 1 yearPopulation: Participants who returned for their one year follow up
The Bayley Scales of Infant and Toddler Development (BSID-III) is designed to assess developmental functioning of infants and toddlers, ages 1 month to 42 months. The instrument includes five distinct scales, of which three scales and associated subscales are utilized for the purposes of this study: cognitive, language (receptive and expressive communication) and motor (fine motor and gross motor). Raw scores are converted to scaled scores using age-standardized norm. The cognitive scaled scores range from 1-19. 1 is a low score and 19 is a high score. The Language scaled scores are calculated by adding the Receptive Communication scores ranging from 1-19 and the Expressive communication scores ranging from 1-19 to give the Language Scaled score of 2-38. The Motor scaled scores are calculated by adding the Fine Motor scores ranging from 1-19 and the Gross Motor scores ranging from 1-19 to give the Motor scaled ranging from 2-38. Higher scores are better than lower scores.
Outcome measures
| Measure |
Reduced Lipid
n=5 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=3 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Bayley Scales for Infant and Toddler Development (BSID-III) at One Year
Cognitive Scaled
|
9 scores on a scale
Interval 7.0 to 14.0
|
10 scores on a scale
Interval 9.0 to 13.0
|
|
Bayley Scales for Infant and Toddler Development (BSID-III) at One Year
Language Scaled
|
18 scores on a scale
Interval 15.0 to 20.0
|
16 scores on a scale
Interval 13.0 to 17.0
|
|
Bayley Scales for Infant and Toddler Development (BSID-III) at One Year
Motor Scaled
|
15 scores on a scale
Interval 15.0 to 20.0
|
20 scores on a scale
Interval 12.0 to 20.0
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Participants who returned for their 2 year follow-up (Note: subjects who did not do their one year follow up were included)
The Bayley Scales of Infant and Toddler Development (BSID-III) is designed to assess developmental functioning of infants and toddlers, ages 1 month to 42 months. The instrument includes five distinct scales, of which three scales and associated subscales are utilized for the purposes of this study: cognitive, language (receptive and expressive communication) and motor (fine motor and gross motor). Raw scores are converted to scaled scores using age-standardized norm. The cognitive scaled scores range from 1-19. 1 is a low score and 19 is a high score. The Language scaled scores are calculated by adding the Receptive Communication scores ranging from 1-19 and the Expressive communication scores ranging from 1-19 to give the Language Scaled score of 2-38. The Motor scaled scores are calculated by adding the Fine Motor scores ranging from 1-19 and the Gross Motor scores ranging from 1-19 to give the Motor scaled ranging from 2-38. Higher scores are better than lower scores.
Outcome measures
| Measure |
Reduced Lipid
n=6 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=2 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Bayley Scales for Infant and Toddler Development (BSID-III) at Two Years
Cognitive Scaled
|
7.5 score on a scale
Interval 3.0 to 9.0
|
10.5 score on a scale
Interval 9.0 to 12.0
|
|
Bayley Scales for Infant and Toddler Development (BSID-III) at Two Years
Language Scaled
|
17 score on a scale
Interval 4.0 to 21.0
|
23 score on a scale
Interval 22.0 to 24.0
|
|
Bayley Scales for Infant and Toddler Development (BSID-III) at Two Years
Motor Scaled
|
17.5 score on a scale
Interval 9.0 to 20.0
|
22 score on a scale
Interval 22.0 to 22.0
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Participants who returned for their 2 year follow-up (Note: subjects who did not do their one year follow up were included)
The MacArthur-Bates Communicative Development Inventories (CDI) are parent report instruments which capture information about children's developing abilities in early language. Scores are reported as percentiles compared to age-standardized norms. Higher scores are better than lower scores.
Outcome measures
| Measure |
Reduced Lipid
n=6 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=2 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
MacArthur-Bates Communicative Development Inventories (CDI)
Vocabulary Percentile
|
25 percentile
Interval 7.0 to 52.5
|
57.5 percentile
Interval 42.5 to 72.5
|
|
MacArthur-Bates Communicative Development Inventories (CDI)
Word Forms Percentile
|
20 percentile
Interval 10.0 to 65.0
|
50 percentile
Interval 35.0 to 65.0
|
|
MacArthur-Bates Communicative Development Inventories (CDI)
Complexity Percentile
|
16.25 percentile
Interval 7.5 to 77.5
|
88.75 percentile
Interval 85.0 to 92.5
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Participants who returned for their 2 year follow-up (Note: subjects who did not do their one year follow up were included)
Dichotomous scores are generated based on cut-off scores, which identify subjects to be at risk. The problem scale measures behaviors of the child that if present, represent a problem. The competence scale measures behaviors of the child that if absent, represent a problem. BITSEA percentile rankings are determined from a table that has a limited range and are adjusted for age and sex. A high problem score leads to a low problem percentile. A high competence score leads to a high competence percentile. Percentile rankings for both problem and competence scores range from "4% or less" to "26% or higher". 4 is the lowest percentile score and 26 is the highest percentile score. The 25th percentile is the lower limit of the average range. Higher percentile scores are better than lower percentile scores in both problem and competence categories.
Outcome measures
| Measure |
Reduced Lipid
n=6 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=2 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Brief Infant Toddler Social Emotional Assessment (BITSEA) Part 1 of 2
Problem Percentile
|
26 percentile
Interval 12.0 to 26.0
|
26 percentile
Interval 26.0 to 26.0
|
|
Brief Infant Toddler Social Emotional Assessment (BITSEA) Part 1 of 2
Competence Percentile
|
25.5 percentile
Interval 4.0 to 26.0
|
26 percentile
Interval 26.0 to 26.0
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Subjects who were evaluated using the GMFCS.
This classification is based on observation with a scale of 1-5. A lower number classification is better than a higher classification, with 1 being the best.
Outcome measures
| Measure |
Reduced Lipid
n=5 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=2 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Gross Motor Function Classification System (GMFCS)
Subjects with a classification of 3
|
0 Participants
|
0 Participants
|
|
Gross Motor Function Classification System (GMFCS)
Subjects with a classification of 1
|
5 Participants
|
2 Participants
|
|
Gross Motor Function Classification System (GMFCS)
Subjects with a classification of 2
|
0 Participants
|
0 Participants
|
|
Gross Motor Function Classification System (GMFCS)
Subjects with a classification of 4
|
0 Participants
|
0 Participants
|
|
Gross Motor Function Classification System (GMFCS)
Subjects with a classification of 5
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Participants who returned for their 2 year follow-up (Note: subjects who did not do their one year follow up were included)
The Behavioral Assessment System for Children-Third Edition is a comprehensive set of forms that helps to understand the behaviors and emotions of children. Scores are reported as T-Scores. T-Scores range from 0-120. In a normative population, the mean of standard scores is 50, and standard deviation is 10. For Externalizing Problems T-Score, Internalizing Problems T-Score, Behavioral Symptoms Index T-Score, Clinical Probability Index T-Score, and Functional Impairment Index T-Score lower scores are better than higher scores. For these categories, higher scores are more problematic with scores between 60-70 regarded as "at risk" and scores 70 and above regarded as clinically significant and requiring further assessment and possible treatment. For Adaptive Skills T-Score, a higher score is better than a lower score. For Adaptive Skills T-Score, lower scores are more problematic with scores between 30-40 regarded as "at risk" and scores at or below 30 regarded as clinically significant.
Outcome measures
| Measure |
Reduced Lipid
n=6 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=2 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 1 of 2
Externalizing Problems T-Score
|
43 T-Score
Interval 38.0 to 66.0
|
55.5 T-Score
Interval 44.0 to 67.0
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 1 of 2
Internalizing Problems T-Score
|
47 T-Score
Interval 41.0 to 56.0
|
48 T-Score
Interval 43.0 to 53.0
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 1 of 2
Behavioral Symptoms Index T-Score
|
48.5 T-Score
Interval 41.0 to 65.0
|
53 T-Score
Interval 45.0 to 61.0
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 1 of 2
Clinical Probability Index T-Score
|
51.5 T-Score
Interval 40.0 to 69.0
|
50.5 T-Score
Interval 40.0 to 61.0
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 1 of 2
Functional Impairment Index T-Score
|
52 T-Score
Interval 42.0 to 61.0
|
52 T-Score
Interval 42.0 to 62.0
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 1 of 2
Adaptive Skills T-Score
|
41 T-Score
Interval 35.0 to 51.0
|
49.5 T-Score
Interval 42.0 to 57.0
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Participants who returned for their 2 year follow-up (Note: subjects who did not do their one year follow up were included)
The Behavioral Assessment System for Children-Third Edition is a comprehensive set of forms that helps to understand the behaviors and emotions of children. For Overall Executive Functioning Index, Attentional Control Index, Behavioral Control Index, and Emotional Control Index, scores are "Not Elevated" or "Elevated". Not Elevated is better than Elevated.
Outcome measures
| Measure |
Reduced Lipid
n=6 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=2 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 2 of 2
Overall Executive Functioning Index · Not Elevated
|
5 Participants
|
1 Participants
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 2 of 2
Overall Executive Functioning Index · Elevated
|
1 Participants
|
1 Participants
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 2 of 2
Attentional Control Index · Not Elevated
|
6 Participants
|
1 Participants
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 2 of 2
Attentional Control Index · Elevated
|
0 Participants
|
1 Participants
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 2 of 2
Behavioral Control Index · Not Elevated
|
5 Participants
|
1 Participants
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 2 of 2
Behavioral Control Index · Elevated
|
1 Participants
|
1 Participants
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 2 of 2
Emotional Control Index · Not Elevated
|
5 Participants
|
1 Participants
|
|
Behavioral Assessment System for Children-Third Edition (BASC3) Part 2 of 2
Emotional Control Index · Elevated
|
1 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 2 yearsPopulation: Participants who returned for their 2 year follow-up (Note: subjects who did not do their one year follow up were included)
Dichotomous scores are generated based on cut-off scores, which identify subjects to be at risk. The problem scale measures behaviors of the child that if present, represent a problem. The competence scale measures behaviors of the child that if absent, represent a problem.
Outcome measures
| Measure |
Reduced Lipid
n=6 Participants
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=2 Participants
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Brief Infant Toddler Social Emotional Assessment (BITSEA) Part 2 of 2
Possible Problem
|
1 Participants
|
0 Participants
|
|
Brief Infant Toddler Social Emotional Assessment (BITSEA) Part 2 of 2
No Problem
|
5 Participants
|
2 Participants
|
|
Brief Infant Toddler Social Emotional Assessment (BITSEA) Part 2 of 2
No Deficit/Delay
|
5 Participants
|
2 Participants
|
|
Brief Infant Toddler Social Emotional Assessment (BITSEA) Part 2 of 2
Possible Deficit/Delay
|
1 Participants
|
0 Participants
|
Adverse Events
Reduced Lipid
Standard Lipid
Serious adverse events
| Measure |
Reduced Lipid
n=12 participants at risk
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=9 participants at risk
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
General disorders
Sepsis
|
0.00%
0/12 • Two years
|
22.2%
2/9 • Two years
|
|
Hepatobiliary disorders
PNAC
|
16.7%
2/12 • Two years
|
33.3%
3/9 • Two years
|
|
Infections and infestations
Necrotizing enterocolitis
|
8.3%
1/12 • Two years
|
11.1%
1/9 • Two years
|
|
Vascular disorders
PICC associated DVT
|
0.00%
0/12 • Two years
|
22.2%
2/9 • Two years
|
|
Gastrointestinal disorders
Bowel obstruction
|
8.3%
1/12 • Two years
|
11.1%
1/9 • Two years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia
|
8.3%
1/12 • Two years
|
0.00%
0/9 • Two years
|
|
Gastrointestinal disorders
Ischemic Bowel Disease
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
General disorders
Hyponatremia
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory Failure
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
Endocrine disorders
Adrenal Insufficiency
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
Blood and lymphatic system disorders
Anemia- Serious
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
Blood and lymphatic system disorders
Coagulopathy
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural Effusion
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
Gastrointestinal disorders
Intestinal Failure- Short Bowel Syndrome
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
General disorders
Inadequate Weight Gain
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
General disorders
Metabolic Acidosis- normal anion gap and bicarbonate loss
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
Other adverse events
| Measure |
Reduced Lipid
n=12 participants at risk
Subjects will receive a minimized dose (1 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
Standard Lipid
n=9 participants at risk
Subjects will receive the standard dose (up to 3 g/kg/day) of the soybean-based lipid component of parenteral nutrition.
Intralipid 20% I.V. Fat Emulsion
|
|---|---|---|
|
Surgical and medical procedures
Line Complication, Removal, or Replacement
|
33.3%
4/12 • Two years
|
22.2%
2/9 • Two years
|
|
General disorders
Bradycardia with Tachypnea
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
General disorders
Hyperglycemia
|
8.3%
1/12 • Two years
|
0.00%
0/9 • Two years
|
|
Cardiac disorders
Tachycardia
|
16.7%
2/12 • Two years
|
0.00%
0/9 • Two years
|
|
General disorders
Hypertension
|
8.3%
1/12 • Two years
|
0.00%
0/9 • Two years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory Failure
|
8.3%
1/12 • Two years
|
0.00%
0/9 • Two years
|
|
Infections and infestations
Oral Thrush
|
8.3%
1/12 • Two years
|
0.00%
0/9 • Two years
|
|
General disorders
Non-severe Anemia
|
0.00%
0/12 • Two years
|
11.1%
1/9 • Two years
|
|
General disorders
Inadequate weight gain- non-severe
|
8.3%
1/12 • Two years
|
0.00%
0/9 • Two years
|
|
General disorders
Enteritis
|
8.3%
1/12 • Two years
|
0.00%
0/9 • Two years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place