Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients

• Status: Completed
• Trial end date: 2015-03-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to (1) demonstrate the efficacy and safety (toxicity) of 25 mg/kg/day of Defibrotide in patients with severe veno-occlusive disease (sVOD) and (2) evaluate serum and endothelial markers of veno-occlusive disease (VOD) through the analysis of blood samples.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Jazz Pharmaceuticals
• Collaborator: FDA Office of Orphan Products Development
• Treatments: Defibrotide

Criteria

Inclusion Criteria:

• Clinical diagnosis of VOD, defined by jaundice (bilirubin >/= 2 mg/dL) and at least 2 of the following clinical findings, by Day+21 post stem cell transplant: ascites; weight gain >/= 5% above baseline weight; hepatomegaly.

• Severe VOD, defined as VOD with multi-organ failure, i.e., presence of one or both of the following, by Day+28 post stem cell transplant: renal or pulmonary dysfunction.

• Provide voluntary written informed consent.

Exclusion Criteria:

• Pre-existing (prior to SCT) cirrhosis

• An alternative diagnosis for weight gain, ascites and jaundice

• Graft-versus-host disease (GVHD) grade B or higher involving liver or gut or grade C or higher involving skin

• Prior solid organ transplant

• Dependent on dialysis prior to and/or at the time of SCT

• Dependent on oxygen supplementation prior to SCT

• Significant acute bleeding or hemodynamic instability

• Requirement for the use of any medications that increase risk of hemorrhage will be excluded from the treatment group