This clinical trial is for males and females with veno-occlusive disease who are scheduled to undergo allogeneic or autologous hematopoietic stem cell transplant (HSCT) and are at high risk or very high risk of developing veno-occlusive disease (VOD). The medicines that are received as part of the transplant therapy can sometimes damage the small blood vessels in the liver.
The study drug, Defibrotide, is a complex form of DNA (a component of genetic material) derived from porcine, or pig tissue. It is prepared in such a way that there is no known risk of microbial infection from the medicine. When administered to humans, defibrotide appears to interact with surface structures, called receptors, on blood vessels that are important in controlling inflammation, blood clotting and scar formation (fibrosis).
The purpose of this study is to compare the study drug, defibrotide with best supportive care, to best supportive care alone in prevention of veno-occlusive disease (VOD).
The study subjects will be randomly assigned, with a 50/50 chance of assignment, to receive defibrotide in addition to best supportive care, or best supportive care medicine(s) alone.
The Defibrotide arm will receive study treatment prior to conditioning and for a minimum of 21 days and up to 30 days post HSCT.
The best supportive care arm will receive treatment prior to conditioning and for 30 days post HSCT.
The weekly follow up period will start post HSCT and last for 180 days post-HSCT.
- Males and females age 18 through 65
- Patient must be scheduled to undergo allogeneic or autologous hematopoietic stem cell transplant (HSCT)
- Detailed eligibility will be reviewed when you contact the study team.