Trial Search Results

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

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.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Jazz Pharmaceuticals

Collaborator: FDA Office of Orphan Products Development


  • Drug: Defibrotide


Phase 3


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

Ages Eligible for Study

N/A - N/A

Genders Eligible for Study


Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
BMT Referrals
Not Recruiting