The 80-patient, multi-center trial will be compared to an historical control group of 80 patients, with survival at day 100 as the primary endpoint. Severe veno-occlusive disease (VOD) is defined as VOD with multiple organ failure – according to the company there are currently no approved treatments for this form of the disease.
The company believes that approximately 80% of patients with VOD die within 100 days of stem cell transplantation without treatment.
The institutional review board (IRB) of the Dana-Farber/Harvard Cancer Center of Boston, is also the IRB for Dana-Farber Cancer Institute, Massachusetts General Hospital, Beth Israel Deaconess Medical Center and The Children’s Hospital. All four of these institutions are expected to participate in the trial.
Work to compile historical control data will begin immediately, and the first patients are expected to be treated by early May 2006.
“As we continue to advance the development of defibrotide, we are increasingly impressed by the consistency of the clinical findings and I am optimistic that we will soon be able to offer an effective treatment option for patients suffering from this otherwise often fatal disease,” commented Dr Laura Ferro, president and CEO of Gentium. “We are pleased to initiate this US phase III study and hope to see compelling clinical results.”
The FDA has granted defibrotide orphan drug status and fast track designation for the treatment of severe VOD.