Compassionate Use of Omegaven IV Fat Emulsion

Not Applicable

Withdrawn

• Conditions: Total Parenteral Nutrition-induced Cholestasis
• Interventions: Drug: Omegaven IV lipid emulsion

• Registration Number: NCT01323907
• Lead Sponsor: Sivan Kinberg

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2011-01
• Study First Submitted: 2011-03-16
• Study First Submitted QC: 2011-03-24
• Study First Posted: 2011-03-28
• Primary Completion: 2019-02-13
• Study Completion: 2019-02-13
• Status Verified: 2020-02
• Last Update Submitted: 2020-02-04
• Last Update Posted: 2020-02-06

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Omegaven	Omegaven IV lipid emulsion	Omegaven IV lipid emulsion administration for infants with life threatening parenteral nutrition associated liver disease

Primary Outcome Measures

Name	Time	Method
Reduction of direct serum bilirubin level (mg/dL)	Up to 6 months from the completion of the study.	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

Name	Time	Method
Essential fatty acid profile	Up to 30 days from therapy completion	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.

Trial Locations

Locations (1)

Columbia Presbyterian Medical Center-Children's Hospital of NY; 🇺🇸 New York, New York, United States