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news.Gentium has initiated a phase II/III trial with its investigational candidate Defibrotide to prevent veno-occlusive disease, a complication of bone marrow and stem cell transplantation, in pediatric patients undergoing chemotherapy or radiation therapy.
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The randomized study will include 270 pediatric patients undergoing stem cell transplantation at 30 clinical sites in Europe and Israel and will evaluate the ability of Defibrotide to prevent veno-occlusive disease (VOD).
The European Group for Blood and Marrow Transplantation (EBMT) is co-sponsoring the study with additional support from the Deutsche Krebshilfe (German Cancer Aid).
Certain chemotherapy and radiation therapies such as those used in stem cell transplantation can damage cells of the blood vessels and produce VOD, a blockage of the small veins of the liver which leads to damage of the liver cells.
Approximately 20% of patients who undergo stem cell transplantation develop VOD and approximately one-third of these patients progress to multiple organ failure. Nearly 80% of patients with severe VOD die within three months. There are currently no approved therapies to treat or prevent the disorder.
“Earlier clinical trials of Defibrotide to treat and prevent VOD have shown encouraging results,” said Dr Laura Ferro, president and CEO of Gentium. “Preventing VOD in stem cell transplant patients is critically important, especially for pediatric patients who are particularly susceptible to developing VOD due to the nature of their treatment regimen.”