Clinical trial NCT01861834: Safer Parenteral Nutrition in Pediatric Short Bowel Syndrome to Decrease Liver Damage
Official title: A Safer Approach to Total Parenteral Nutrition in Pediatric Short Bowel Syndrome Intended to Decrease the Frequency and Severity of Liver Damage
Study type: EXPANDED_ACCESS
Overall status: APPROVED_FOR_MARKETING
Conditions: Cholestasis of Parenteral Nutrition
Keywords: Total parenteral nutrition
TPN
Intestinal Failure
Short Bowel Syndrome
Cholestasis
Intestinal failure associated liver disease
Parenteral nutrition associated liver disease
Lead sponsor: Georgetown University
Sponsor class: OTHER
First posted: 2013-05-24
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT

Brief summary: To provide children dependent on total parenteral nutrition with Omegaven®, a fish oil-based intravenous lipid emulsion that may be less hepatotoxic than conventional, vegetable oil-based intravenous lipid emulsions, and that may therefore reduce the need for liver transplantation.


Eligibility criteria: Inclusion Criteria:

* The targeted population for enrollment is the cohort of patients with TPN-dependent short bowel syndrome, defined as any pediatric patient who, following abdominal surgery, has a residual small bowel length less than 25% of that predicted for gestational age or requires postoperative TPN for more than 42 days because of gastrointestinal intolerance and who has developed TPN-associated liver disease sufficient to pose a significant risk for progression to liver failure based on the following criteria:

  * Total serum bilirubin concentration greater than 3 mg/dL after a total duration of TPN greater than 2 months in the absence of a proven episode of bacteremia within the preceding 3 weeks.

PLUS EITHER:

• Platelet count less than 200,000/μL after a total duration of TPN greater than 2 months in the absence of a proven episode of bacteremia within the preceding 3 weeks.

OR

• Serum albumin concentration less than 3.2 mg/dL after a total duration of TPN greater than 2 months in the absence of a proven episode of bacteremia within the preceding 3 weeks.

Patients with coagulopathy due to parenteral nutrition-associated liver disease (INR \> 1.2) will be potential candidates for enrollment, because patients with an elevated INR exceeding 2 have demonstrated resolution of coagulopathy after treatment with Omegaven®. Similarly, patients with hyperlipidemia will be potential candidates for enrollment.

Alternatively, patients currently receiving Omegaven that was initiated at another center because of intestinal failure with liver disease that do not need to meet the lab criteria listed above. The subject may continue Omegaven under this protocol at the discretion of the Principle Investigator.

Exclusion Criteria:

Patients with a history of the following will be excluded from enrollment in this protocol:

* Allergy to fish or egg protein.
* Liver disease proven or suspected to be caused by a process other than TPN-dependent short bowel syndrome, including but not limited to hepatitis C, hepatitis B, cystic fibrosis, biliary atresia, Alagille syndrome, familial intrahepatic cholestasis, and alpha-1-antitrypsin deficiency.
* Refusal of third party providers to reimburse hospital for the cost of Omegaven®.

Primary outcomes:
Secondary outcomes:
Other outcomes:
Locations:
city: Washington
country: United States
facility: MedStar Georgetown Transplant Institute
state: District of Columbia
city: Washington
country: United States
facility: MedStar Georgetown University Hospital
state: District of Columbia
Study officials:
References: