DE0605: Defibrotide for Patients with Hepatic Veno-Occlusive Disease (VOD): A Treatment IND study


Stem Cell / Bone Marrow Transplant : Gene therapy: hepatic veno-occlusive disease


Hepatic veno-occlusive disease (VOD) is a disease when the blood vessels that carry blood through the liver become swollen and blocked causing the liver to not remove toxins, drugs and other waste products from the bloodstream. Hepatic VOD, is one of the most serious and common complications that arises in patients who have undergone high dose chemotherapy exposure followed by Hematopoietic Stem Cell Transplantation (transplantation of blood stem cells derived from the bone marrow or blood). VOD develops in 10-60% of patients after hematopoietic stem cell transplant and ranges in severity from mild, reversible disease to a severe syndrome associated with multi-organ failure and death.

The medication Defibrotide is obtained from the tissue of pigs (a “porcine” based medication). The manufacturer prepares the medication to assure that there are no germs in the product that could be transmitted to the recipient. It is believed that Defibrotide works by preventing the blood from clotting and the blood vessels from becoming swollen and “inflamed.” Results from early human studies suggest that Defibrotide may help to decrease or resolve hepatic VOD.


This research study is evaluating an investigational drug called Defibrotide as a possible treatment for hepatic VOD.This means that Defibrotide is not approved by the U.S. Food and Drug Administration for use outside of a research study.

The primary (main) goal of this study is to provide Defibrotide to patients with VOD.  At present there is no known curative therapy for this disorder. This study seeks to better understand the effects (good and bad) of Defibrotide in patients with severe VOD.


For the current eligibility status of this clinical study, referring physicians must contact St. Jude Children's Research Hospital at 1-866-2ST-JUDE  (1-866-278-5833).


Brandon Triplett, MD
St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN  38105  USA
Phone: 1-866-2ST-JUDE (1-866-278-5833)
FAX: 901-595-5068

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