Compassionate Use of Omegaven in Children

NCT02370251 | Completed Phase 2 Results DMC

• 1 other identifier: 5451
• Study Type: interventional
• Target: 63
• Locations: 1 country
• Sites: 1

Brief Summary

This is a single-assignment study to evaluate whether Omegaven (IV fish oil) is effective at treating liver disease in children on long-term IV nutrition.

Conditions

Total Parenteral Nutrition-induced Cholestasis; Cholestasis; Short Bowel Syndrome; Gastroschisis; Intestinal Atresia; Infant, Premature, Diseases; Cholestasis of Parenteral Nutrition

Keywords

Fat Emulsions, Intravenous; Parenteral-Nutrition-Associated Liver Disease; Cholestasis; Omegaven; Fish Oil

Outcome Measures

Primary Outcomes (1)

• Resolution of Cholestasis for Subjects Who Received Omegaven

  To determine if Omegaven results in the resolution of cholestasis (DB \<2 for 2 consecutive weeks)

  Within the first 3 months of sole Omegaven use

Secondary Outcomes (2)

• Safety Issues for Infants Who Received Omegaven

  Within the first year of use

• Essential Fatty Acid Deficiency in Infants Who Received Omegaven

  Within the first month of use

Study Arms (1)

Omegaven

EXPERIMENTAL

Children will receive Omegaven at a maximum of 1 g/kg/day upon enrollment in this arm.

Drug: Omegaven

Interventions

Omegaven DRUG

Once the direct bilirubin is 2 mg/dL or more x 2 weeks, Intralipid will be switched to Omegaven at 1 g/kg/day. The bilirubin level will be monitored to determine when resolution of cholestasis (DB \<2 mg/dL) occurs.

Also known as: Fish oil-based lipid, Omega-3-fatty acid lipid emulsion

Omegaven

Eligibility Criteria

• Age: Up to 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Live in or temporarily relocate to Oklahoma
• Age less than 18 years, both sexes, all races
• Have a direct bilirubin level of ≥2 mg/dL for two consecutive weeks after at least 14 days of parenteral nutrition
• Received parenteral lipids at a maximum dose of 1.7 g/kg/day (24 g/kg over the two weeks prior)
• Are not currently enrolled in another lipid emulsion study

You may not qualify if:

• Known food allergy to fish
• Known metabolic disorder of lipid metabolism
• Active coagulopathies (active bleeding or requiring blood product treatment in the prior 48 hours)
• Medical condition likely to result in death in the next 30 days

Sponsors & Collaborators

• University of Oklahoma: lead
• OU Medical Center: collaborator

Study Sites (1)

OU Children's Hospital

Oklahoma City, Oklahoma, 73104, United States

Location

MeSH Terms

Conditions

Cholestasis; Short Bowel Syndrome; Gastroschisis; Intestinal Atresia; Infant, Premature, Diseases

Interventions

fish oil triglycerides

Condition Hierarchy (Ancestors)

Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases; Malabsorption Syndromes; Intestinal Diseases; Gastrointestinal Diseases; Postoperative Complications; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Musculoskeletal Abnormalities; Musculoskeletal Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hernia, Abdominal; Hernia; Pathological Conditions, Anatomical; Digestive System Abnormalities; Infant, Newborn, Diseases

Limitations and Caveats

This was a compassionate use study, using Omegaven as a "last resort" for infants with a direct bilirubin of at least 2 mg/dL at the time of initiation, so these infants were already at higher risk of death and hematological/hepatobiliary morbidity.

Results Point of Contact

• Title: Kimberly Ernst, MD
• Organization: University of Oklahoma

kimberly-ernst@ouhsc.edu

405-271-5215

Study Officials

• Kimberly D Ernst, MD, MSMI

  The University of Oklahoma, Department of Pediatrics

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 17, 2015
• First Posted: February 24, 2015
• Study Start: June 1, 2015
• Primary Completion: September 27, 2018
• Study Completion: September 27, 2019
• Last Updated: November 25, 2019
• Results First Posted: November 25, 2019

Record last verified: 2019-11

Locations

US (1)