Severe Hepatic Veno Occlusive Disease Clinical Trial
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
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 VOD through the analysis of blood samples.
This is a historically-controlled, multicenter, open label Phase 3 study to determine the
safety and efficacy of 25 mg/kg/day of Defibrotide (DF) for the treatment of severe VOD in
hematopoietic SCT patients.
In this study, the term "severe VOD" is defined as those patients who meet the Baltimore
diagnostic criteria for VOD (total bilirubin >/= 2.0 mg/dL plus two of the following:
ascites, >/=5% weight gain and hepatomegaly), who also have MOF (i.e., pulmonary and/or
renal dysfunction). This represents a group of patients in whom mortality at D+100 has been
estimated to be >80%.
Comparisons: The primary parameter is Complete Response at 100 days following stem cell
transplant, utilizing historical controls as a comparator. The historical control database
will be generated through a retrospective medical chart review performed at participating
centers; the survival outcome of patients who would otherwise have met eligiblity criteria
for this trial will be compared to the survival observed in patients prospectively treated
with defibrotide. Secondary parameters include survival rate at 100 days and 6 months post
SCT, and special studies of endothelial and serum markers for VOD. This study will assess
safety of the dose and schedule in this setting.
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Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment