Back to resultsCompassionate Use of Omegaven IV Fat Emulsion
Primary Purpose
Total Parenteral Nutrition-induced Cholestasis
Status
Withdrawn
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
Omegaven IV lipid emulsion
Sponsored by
About this trial
This is an interventional treatment trial for Total Parenteral Nutrition-induced Cholestasis focused on measuring Total Parenteral Nutrition-induced Cholestasis, Omegaven, liver disease, Cholestasis, Fat emulsions
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of life threatening PNALD
- Dependent on Parenteral Nutrition for caloric needs
- Expected to require PN for at least another 30 days
- Other causes of liver disease have been excluded
- GI/Liver service is involved in patient care
- Standard therapies for the treatment of PNALD must have been utilized prior to initiating omegaven.
- Inpatient at Morgan Stanley Children's Hospital of NewYork Presbyterian Hospital
- Must be at least 2 months of age
Exclusion Criteria:
- Not inpatient
- Younger than 2 months of age
- Expected to be weaned off of parenteral nutrition within 30 days
- have other documented causes of liver disease
- have signs of proven severe advanced liver disease
- Allergy to seafood, egg protein and/or previous allergy to Omegaven
- active coagulopathy, impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis, stoke, embolism, hemodynamic collapse or shock, recent MI, cholestasis due to any reason other than PNALD, active new infection at time of initiation of Omegaven, hemodynamic instability or if unable to tolerate the necessary laboratory monitoring
- must not be enrolled in another clinical trial involving an investigational agent (unless approved by the designated physicians).
- Parent or legal guardian must be willing to provide consent.
Sites / Locations
- Columbia Presbyterian Medical Center-Children's Hospital of NY
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Omegaven
Arm Description
Omegaven IV lipid emulsion administration for infants with life threatening parenteral nutrition associated liver disease
Outcomes
Primary Outcome Measures
Reduction of direct serum bilirubin level (mg/dL)
For effectiveness endpoint, the allowable minimum time interval between serial direct bilirubin measurements that will determine reversal of cholestasis is 2 consecutive measurements of direct bilirubin < or = to 2mg/dL obtained at least 2 weeks apart.
Secondary Outcome Measures
Essential fatty acid profile
Previous studies have found no association between Omegaven administration and essential fatty acid deficiency. Due to theoretical concern for developing fatty acid deficiency with low dose Omegaven administration, these levels will be followed during Omegaven therapy.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01323907
Brief Title
Compassionate Use of Omegaven IV Fat Emulsion
Official Title
Compassionate Use of Omegaven IV Fat Emulsion in Infants With Life Threatening Parenteral Nutrition-associated Liver Disease (PNALD).
Study Type
Interventional
2. Study Status
Record Verification Date
February 2020
Overall Recruitment Status
Withdrawn
Why Stopped
Omegaven is now FDA approved.
Study Start Date
January 2011 (Anticipated)
Primary Completion Date
February 13, 2019 (Actual)
Study Completion Date
February 13, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor-Investigator
Name of the Sponsor
Sivan Kinberg
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of the study is to provide use of Omegaven in children with life threatening Total Parenteral Nutrition (TPN) associated cholestasis when all other medical and surgical treatments/therapies have been either ineffective or not feasible in the treatment of this serious condition. This is a compassionate use protocol.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Total Parenteral Nutrition-induced Cholestasis
Keywords
Total Parenteral Nutrition-induced Cholestasis, Omegaven, liver disease, Cholestasis, Fat emulsions
7. Study Design
Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Omegaven
Arm Type
Experimental
Arm Description
Omegaven IV lipid emulsion administration for infants with life threatening parenteral nutrition associated liver disease
Intervention Type
Drug
Intervention Name(s)
Omegaven IV lipid emulsion
Other Intervention Name(s)
Omega-3 Fatty Acids
Intervention Description
For infants meeting inclusion criteria and whose guardians consent to participation, Omegaven will be initiated at the dose of 0.5gram/kg/day and is infused over 24 hours for 1-2 days and then advanced to 1 gram/kg/day. Omegaven will be infused intravenously through a central or peripheral catheter alone or in conjunction with parenteral nutrition.
Primary Outcome Measure Information:
Title
Reduction of direct serum bilirubin level (mg/dL)
Description
For effectiveness endpoint, the allowable minimum time interval between serial direct bilirubin measurements that will determine reversal of cholestasis is 2 consecutive measurements of direct bilirubin < or = to 2mg/dL obtained at least 2 weeks apart.
Time Frame
Up to 6 months from the completion of the study.
Secondary Outcome Measure Information:
Title
Essential fatty acid profile
Description
Previous studies have found no association between Omegaven administration and essential fatty acid deficiency. Due to theoretical concern for developing fatty acid deficiency with low dose Omegaven administration, these levels will be followed during Omegaven therapy.
Time Frame
Up to 30 days from therapy completion
10. Eligibility
Sex
All
Minimum Age & Unit of Time
2 Months
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of life threatening PNALD
Dependent on Parenteral Nutrition for caloric needs
Expected to require PN for at least another 30 days
Other causes of liver disease have been excluded
GI/Liver service is involved in patient care
Standard therapies for the treatment of PNALD must have been utilized prior to initiating omegaven.
Inpatient at Morgan Stanley Children's Hospital of NewYork Presbyterian Hospital
Must be at least 2 months of age
Exclusion Criteria:
Not inpatient
Younger than 2 months of age
Expected to be weaned off of parenteral nutrition within 30 days
have other documented causes of liver disease
have signs of proven severe advanced liver disease
Allergy to seafood, egg protein and/or previous allergy to Omegaven
active coagulopathy, impaired lipid metabolism or severe hyperlipidemia with or without pancreatitis, stoke, embolism, hemodynamic collapse or shock, recent MI, cholestasis due to any reason other than PNALD, active new infection at time of initiation of Omegaven, hemodynamic instability or if unable to tolerate the necessary laboratory monitoring
must not be enrolled in another clinical trial involving an investigational agent (unless approved by the designated physicians).
Parent or legal guardian must be willing to provide consent.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sivan Kinberg, MD, MS
Organizational Affiliation
Columbia University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Columbia Presbyterian Medical Center-Children's Hospital of NY
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Compassionate Use of Omegaven IV Fat Emulsion
We'll reach out to this number within 24 hrs