Trial Outcomes & Findings for Compassionate Use of Omegaven to Reverse Parenteral Nutrition Induced Cholestasis (NCT NCT01173159)
NCT ID: NCT01173159
Last Updated: 2020-06-17
Results Overview
Change in conjugated/direct bilirubin level to below 1 mg/dl.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
10 participants
Primary outcome timeframe
Completion of Therapy (time frame from 1-14 weeks)
Results posted on
2020-06-17
Participant Flow
Participant milestones
| Measure |
Omegaven
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require Total Parenteral Nutrition or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
10
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Compassionate Use of Omegaven to Reverse Parenteral Nutrition Induced Cholestasis
Baseline characteristics by cohort
| Measure |
Omegaven
n=10 Participants
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require TPN or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
Age, Continuous
|
6 months
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 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
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
6 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
|
PRIMARY outcome
Timeframe: Completion of Therapy (time frame from 1-14 weeks)Change in conjugated/direct bilirubin level to below 1 mg/dl.
Outcome measures
| Measure |
Omegaven
n=10 Participants
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require TPN or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
Number of Participants With a Change in Conjugated/Direct Bilirubin
|
4 Participants
|
SECONDARY outcome
Timeframe: Completion of Therapy (time frame from 1-14 weeks)Change in unconjugated/total bilirubin level to below 1.1 mg/dL. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Outcome measures
| Measure |
Omegaven
n=10 Participants
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require TPN or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
Number of Participants With a Change in Unconjugated/Total Bilirubin
|
5 Participants
|
SECONDARY outcome
Timeframe: Completion of Therapy (time frame from 1-14 weeks)Change in aspartate transaminase (AST) 57 units/L. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Outcome measures
| Measure |
Omegaven
n=10 Participants
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require TPN or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
Number of Participants With a Change in Aspartate Transaminase (AST)
|
2 Participants
|
SECONDARY outcome
Timeframe: Completion of Therapy (time frame from 1-14 weeks)Change in liver enzyme ALT to below 59 unit/L. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Outcome measures
| Measure |
Omegaven
n=10 Participants
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require TPN or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
Number of Participants With a Change in Liver Enzyme (ALT)
|
1 Participants
|
SECONDARY outcome
Timeframe: Completion of Therapy (time frame from 1-14 weeks)Change in liver enzyme alkaline phosphatase to below 345 unit/L. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Outcome measures
| Measure |
Omegaven
n=10 Participants
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require TPN or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
Number of Participants With a Change in Liver Enzyme Alkaline Phosphatase
|
3 Participants
|
SECONDARY outcome
Timeframe: Completion of Therapy (time frame from 1-14 weeks)Change in Liver Enzyme Gamma-glutamyltransferase (GGT) to below 15 unit/L. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Outcome measures
| Measure |
Omegaven
n=10 Participants
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require TPN or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
Number of Participants With a Change in Liver Enzyme Gamma-glutamyltransferase (GGT)
|
0 Participants
|
SECONDARY outcome
Timeframe: Completion of Therapy (time frame from 1-14 weeks)Change in Triglycerides to below 119 mg/dL. General outcomes of participants are reported due to the enrollment of very few patients so that we have too small a cohort with which to assess achievement of our stipulated outcomes.
Outcome measures
| Measure |
Omegaven
n=10 Participants
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require TPN or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
Number of Participants With a Change in Triglycerides
|
8 Participants
|
Adverse Events
Omegaven
Serious events: 4 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Omegaven
n=10 participants at risk
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require TPN or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
Blood and lymphatic system disorders
Sepsis
|
10.0%
1/10 • Number of events 2 • 1 to 14 weeks of therapy
|
|
Renal and urinary disorders
Acute Kidney Injury
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Injury, poisoning and procedural complications
Compartment Syndrome Following Surgery
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Cardiac disorders
Sinus Bradycardia
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Cardiac disorders
Hyperkalemia Causing Cardiac Dysrhythmia
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Cardiac disorders
Cardiac Arrest
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Gastrointestinal disorders
Gastrointestinal Hemorrhage
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Congenital, familial and genetic disorders
Multisystem Organ Dysfunction related to IPEX
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
Other adverse events
| Measure |
Omegaven
n=10 participants at risk
Subjects will receive Omegaven at a dose of up to 1 g/kg body weight/day until they no longer require TPN or until their conjugated/direct bilirubin has normalized and their enteral lipid intake is sufficient to discontinue intravenous lipids.
Omegaven: For the first two days of treatment, subjects will receive Omegaven® at 0.5 g/kg per day to assess tolerance and will progress to a maintenance dosage of up to 1g/kg per day over 12 hours at an infusion rate of 1 g/kg/12 hours (10 ml/kg/12 hours). Dosing is based on previously described dosing of fish-oil emulsions as monotherapy noted within the literature. Omegaven® will be infused intravenously through either a central or peripheral catheter in conjunction with other parenteral nutrition containing dextrose and amino acids. Omegaven® is isotonic. It is compatible with parenteral nutrition solutions and may be co-infused via y-site.
|
|---|---|
|
General disorders
Vomiting
|
20.0%
2/10 • Number of events 3 • 1 to 14 weeks of therapy
|
|
General disorders
Fever
|
30.0%
3/10 • Number of events 6 • 1 to 14 weeks of therapy
|
|
General disorders
Upper Respiratory Infection
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Investigations
Phosporus Decreased
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Skin and subcutaneous tissue disorders
Skin Irritation and breakdown
|
20.0%
2/10 • Number of events 2 • 1 to 14 weeks of therapy
|
|
Skin and subcutaneous tissue disorders
Diaper Dermatitis
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Vascular disorders
Pulmonary Vein Stenosis
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Gastrointestinal disorders
Gastrointestinal Fistula
|
20.0%
2/10 • Number of events 2 • 1 to 14 weeks of therapy
|
|
Gastrointestinal disorders
Blood in Stool
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Blood and lymphatic system disorders
Edema
|
20.0%
2/10 • Number of events 2 • 1 to 14 weeks of therapy
|
|
Gastrointestinal disorders
Portal Hypertension
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Cardiac disorders
Ventricular Tachycardia
|
10.0%
1/10 • Number of events 2 • 1 to 14 weeks of therapy
|
|
General disorders
Irritability
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
General disorders
Dried blood near Anus
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Cardiac disorders
Tachycardia and Tachypnea
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Infections and infestations
Culture Positive from Tracheostomy
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Blood and lymphatic system disorders
Triglycerides Increased
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Hepatobiliary disorders
Gull bladder sludge with intrahepatice biliary ductal dilation
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Blood and lymphatic system disorders
Activated Partial Throboplastin Time Prolonged
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
General disorders
Lethargy
|
10.0%
1/10 • Number of events 2 • 1 to 14 weeks of therapy
|
|
Blood and lymphatic system disorders
Platelet Count Decreased
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
General disorders
Nasal Congestion
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Respiratory, thoracic and mediastinal disorders
Tachypnea
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
General disorders
Cough
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Respiratory, thoracic and mediastinal disorders
Decreased Respiratory Rate
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Hepatobiliary disorders
Bleeding from nose
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
|
Blood and lymphatic system disorders
Abnormal CBC Differential with Active Bleeding
|
10.0%
1/10 • Number of events 1 • 1 to 14 weeks of therapy
|
Additional Information
Crystal Slaughter, BA, CCRC
Cincinnati Children's Hospital Medical Center
Phone: 513-636-0137
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place