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

Trial ID or NCT#

NCT00358501

Status

not recruiting iconNOT RECRUITING

Purpose

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.

Official Title

Defibrotide for the Treatment of Severe Hepatic Veno-Occlusive Disease in Hematopoetic Stem Cell Transplant Patients: A Historically-Controlled, Multi-Center Phase 3 Study to Determine Safety & Efficacy

Eligibility Criteria

Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
Inclusion Criteria:
  1. - 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:
  1. - 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
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Investigator(s)

Sally Arai
Sally Arai
Blood and marrow transplant specialist, Hematologist, Blood and marrow transplant specialist
Associate Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)
Robert Lowsky
Robert Lowsky
Blood and marrow transplant specialist, Hematologist, Blood and marrow transplant specialist
Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)
Laura Johnston
Laura Johnston
Blood and marrow transplant specialist, Blood and marrow transplant specialist, Medical oncologist, Hematologist
Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)
Robert Negrin
Robert Negrin
Blood and marrow transplant specialist, Hematologist, Blood and marrow transplant specialist
Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy)

Contact us to find out if this trial is right for you.

Contact

BMT Referrals
6507230822

View on ClinicalTrials.gov