Efficacy and Safety of Omega-3 Lipid Therapy in Pediatric Patients With Parenteral Nutrition-Associated Liver Disease

Amarnath, Rathna, M.D.

Status and phase: Unknown; Phase 2

Conditions: Short Bowel Syndrome; Cholestasis

Treatments: Drug: Omegaven Therapy

Study type: Interventional

Identifiers

NCT01739517

IND104555

Details and patient eligibility

About

This pilot study seeks to demonstrate the efficacy of an intravenous lipid preparation high in omega-3 fatty acids (Omegaven) in the treatment of cholestasis in parenteral nutrition dependent patients with short gut syndrome.

Enrollment: 20 estimated patients

Sex: All

Ages: Under 1 year old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patient will be dependent upon parenteral nutrition (PN)
• Patient will have short gut syndrome (loss of >50% of small bowel)
• Patient's guardian/caregiver provides informed consent for patient to receive therapy
• Pediatric patient ≤ 1 year of age
• Expected PN duration is greater than 30 days
• Direct bilirubin >2.0 mg/dL measured on two occasions no more than one week apart

Exclusion criteria

• Liver dysfunction secondary to cause other than PN verified by standard of care diagnostic procedures and lab work to rule out alternative causes of neonatal cholestasis.
• Any patient in whom Omegaven therapy would be contraindicated, such as an allergy to any seafood product, egg protein, and/or previously established allergy to Omegaven
• impaired lipid metabolism
• severe hemorrhagic disorder
• unstable diabetes mellitus
• collapse and shock, stroke/embolism, recent cardiac infarction, or undefined coma status

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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20 participants in 1 patient group

Omegaven Therapy

Experimental group

Description:

After baseline labs, which have been collected no earlier than seven days prior to the initiation of therapy are obtained, therapy with Omegaven will be initiated at a starting dose of 0.5 g/kg/day infused over 12 hours. If tolerated, the dose will be increased to 1 g/kg/day, the goal dose. Omegaven will be infused intravenously through either a central or peripheral catheter in conjunction with parenteral nutrition.

Treatment:

Drug: Omegaven Therapy