Pfizer COVID-19 vaccine appointments are available to our patients. Sign up for Connect today to schedule your vaccination.

A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant

Clinical Trial Details

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.

Key Eligibility: 

   - 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.

Study contact by location

Upper East Side - Manhattan


Ashlee N. Torres, RN
(212) 746-7117

Primary Investigator(s)

Protocol ID(s)

Weill Cornell Medicine IRB #:




Jazz 15-007


Open to Enrollment

Age Group