Gentium gets board OK to start phase 3 trial of Defibrotide for veno-occlusive disease

By Lisa Kerner

Erie, Pa., March 28 - Gentium SpA said it received its first Institutional Review Board approval to begin a phase 3 clinical trial with Defibrotide for the treatment of veno-occlusive disease (VOD) with multiple organ failure (severe VOD) as a complication of stem-cell transplantation.

Data from the 80-patient, multi-center trial will be compared to an historical control group of 80 patients, according to a company news release. The primary endpoint is survival at day 100.

Gentium believes that about 80% of patients with severe VOD die within 100 days of stem-cell transplantation without treatment.

The multi-center trial will be conducted at about 20 U.S. cancer centers pending the Institutional Review Board approval at each institution. The first patients are expected to be treated by early May, the company said.

"Treatment of severe VOD with Defibrotide resulted in a survival rate at 100 days post [stem-cell transplantation] of approximately 40%, which compares favorably with the 100-day survival rate of approximately 20% or less reported in published studies of severe VOD," the principal investigator of the phase 3 trial, Paul Richardson, said in the release.

"These results are encouraging, with response and long-term survival seen even in patients who either were on dialysis or were ventilator-dependent at the start of Defibrotide therapy. In addition, the side effect profile of Defibrotide was favorable in this extremely sick patient population."

The Food and Drug Administration has granted Defibrotide orphan drug status and fast-track designation for the treatment of severe VOD.

VOD is a blockage of the small veins of the liver that can lead to liver and other organ failure. It can be caused by certain high-dose chemotherapy, radiation and stem cell transplantation that damages blood vessel cells.

According to Gentium, about 20% of patients who undergo stem-cell transplantation develop VOD, and about one-third of those who develop VOD progress to severe VOD.

Gentium is a biopharmaceutical company located in Villa Guardia, Italy, focused on the research, discovery and development of drugs to treat and prevent a variety of vascular diseases and conditions related to cancer and cancer treatments.

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