Compassionate Use of an Intravenous Fish Oil Emulsion in the Treatment of Liver Injury in Infants

NCT00738101 | Completed Results DMC

• 1 other identifier: H-23365
• Study Type: interventional
• Target: 293
• Locations: 1 country
• Sites: 2

Brief Summary

To provide a mechanism for critically ill infants with parenteral nutrition (PN) associated cholestasis to receive Omegaven for compassionate use situations for which there are no satisfactory alternative treatments.

Conditions

Cholestasis; Cholestasis of Parenteral Nutrition

Keywords

Omega-3; Omegaven; Cholestasis; Liver damage

Outcome Measures

Primary Outcomes (3)

• Time to Resolution of Parenteral Nutrition Associated Cholestasis Prior to End of Study

  Time in days from the initiation of fish oil emulsions (initiation of study) until resolution of cholestasis as defined by serum conjugated bilirubin≤ 2 mg/dL prior to EOS (end of study).

  From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).

• All Cause Mortality During the Study.

  To describe proportion of infants who died secondary to any cause, related or unrelated to Fish Oil Emulsion.

  From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).

• Growth Z-scores for Weight

  The Z-score indicated the number of standard deviations away from the mean. A weight Z-score of 0 is equal to the mean. A weight Z-score of ≤ -2 indicates an underweight status, while a weight Z-score of ≥ 2 indicates overweight or obese status.

  From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).

Secondary Outcomes (6)

• Number of Infants Who Achieve Resolution of Parenteral Nutrition Associated Cholestasis

  From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).

• Number of Subjects With Platelet Count <100,000/µL

  From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).

• Number of Subjects With INR ≥1.4.

  From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).

• Number of Study Subjects Who Experienced a Blood Stream Infection

  From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).

• Liver or Multi-visceral Transplant

  From initiation to end of study (End of Study : Discontinuation of Fish Oil Emulsion, Death, Transplant, or Discharge from the hospital, whichever is achieved first, up to 5 years).

Study Arms (1)

Fish Oil Emulsion Arm

EXPERIMENTAL

In infants who meet the eligibility criteria for Fish Oil Emulsion arm will receive Fish Oil Emulsion after enrollment under the study. Therapy with Fish Oil Emulsion (Omegaven) will be provided at a dose of 1 gm/kg/day (by continuous infusion) and will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition. If previously on Intralipid, it will be stopped prior to initiation of Fish Oil Emulsion.Fish oil emulsion will be provided as a continuous intravenous emulsion over 24 hours.

Drug: Omegaven

Interventions

Omegaven DRUG

Therapy with Omegaven will be provided at a dose of 1 gm/kg/day (by continuous infusion). Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition. Treatment will be given for as long as the child needs any TPN AND has a conjugated bilirubin greater than 2 mg/dL for a maximum of 5 years. If the infant no longer is requiring any TPN, then the Omegaven will be stopped regardless of bilirubin. If the bilirubin is less than 2 mg/dL but the child still requires TPN, then the Omegaven will be continued until the infant no longer requires TPN.

Also known as: Omega-3 enriched fat emulsion

Fish Oil Emulsion Arm

Eligibility Criteria

• Age: 14 Days - 5 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Be greater than 14 days old and less than 5 years old
• Conjugated bilirubin greater than 2 mg/dL.
• Be expected to require intravenous nutrition for at least an additional 28 days

You may not qualify if:

• Have a congenitally lethal condition (e.g. Trisomy 13).
• Have clinically severe bleeding not able to be managed with routine measures.
• Have evidence of a viral hepatitis or primary liver disease as the primary etiology of their cholestasis.
• Have other health problems such that survival is extremely unlikely even if the infant's cholestasis improves.
• Home Use of Omegaven®:
• In order for a subject to receive the Omegaven® at home through a home health care agency, subjects will first be required to be admitted to Texas Children's Hospital for 72 hours in initiate the administration of the Omegaven®. This will allow time for observation of any unexpected side effects and for parents to be provided education on home TPN and Omegaven®.
• If a subject has already received Omegaven® either at TCH or at another hospital, they will not be required to be admitted for the 72 hour inpatient admission prior to starting Omegaven® at home. Parent training will occur during the previous hospital admission and will continue through the TCH Pediatric Intestinal Rehabilitation Clinic.
• Outpatient Monitoring:
• After the initial evaluation by the TCH Pediatric Intestinal Rehabilitation Clinic physicians, subjects will return to the clinic for routine follow-up. Subjects will be asked to return to the clinic every 2 weeks for the first 2 months of treatment. Thereafter, subjects will return to the clinic on a monthly basis, or as directed by the clinic team.

Sponsors & Collaborators

• Baylor College of Medicine: lead
• Boston Children's Hospital: collaborator

Study Sites (2)

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Related Publications (27)

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• Gura KM, Premkumar MH, Calkins KL, Puder M. Fish Oil Emulsion Reduces Liver Injury and Liver Transplantation in Children with Intestinal Failure-Associated Liver Disease: A Multicenter Integrated Study. J Pediatr. 2021 Mar;230:46-54.e2. doi: 10.1016/j.jpeds.2020.09.068. Epub 2020 Oct 8.

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MeSH Terms

Conditions

Cholestasis

Interventions

fish oil triglycerides

Condition Hierarchy (Ancestors)

Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Limitations and Caveats

The study protocol was terminated early following the FDA approval of the Fish Oil Emulsion (Omegaven) as as source of nutrition in infants with parenteral nutrition associated cholestasis.

Results Point of Contact

• Title: Dr. Muralidhar H Premkumar
• Organization: Baylor College of Medicine

premkuma@bcm.edu

8328267980

Study Officials

• Murali Premkumar, MD

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Masking Details: Open Label
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Assistant Professor

Model Details: Initiation of Study is defined as the day the Fish Oil Emulsion was initiated. End of Study (EOS) is defined as discontinuation of fish oil emulsion, death, transplant, or discharge from the hospital. In infants who meet the eligibility criteria for Fish Oil Emulsion arm will receive Fish Oil Emulsion after enrollment under the study. Therapy with Fish Oil Emulsion (Omegaven) will be provided at a dose of 1 gm/kg/day (by continuous infusion) and will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition. If previously on Intralipid, it will be stopped prior to initiation of Fish Oil Emulsion.

Study Record Dates

• First Submitted: August 18, 2008
• First Posted: August 20, 2008
• Study Start: September 1, 2008
• Primary Completion: March 27, 2019
• Study Completion: March 27, 2020
• Last Updated: May 28, 2021
• Results First Posted: May 21, 2020

Record last verified: 2021-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)