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
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.
Interventional
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Complete Response at D+100 post stem cell transplant
Day + 100 post stem cell transplant
No
Paul Richardson, M.D.
Principal Investigator
Dana-Farber Cancer Institute
United States: Food and Drug Administration
2005-01
NCT00358501
July 2006
November 2008
Name | Location |
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Hinsdale, Illinois 60521 | |
Alexandria, Minnesota 56308 | |
Albany, Georgia 31701 | |
Fountain Valley, California 92708 | |
Columbia, Missouri 65203 | |
Albany, New York 12208 | |
Cleveland, Ohio 44195 | |
Philadelphia, Pennsylvania 19104 | |
Austin, Texas 78705 | |
Seattle, Washington 98195 | |
Flint, Michigan 48532 | |
Omaha, Nebraska 68114 | |
Hackensack, New Jersey 07601 | |
Denver, Colorado | |
Baltimore, Maryland 21287 | |
Boston, Massachusetts | |
Charlotte, North Carolina | |
Eugene, Oregon | |
Indianapolis, Indiana |