Gentium starts phase 2/3 pediatric trial with Defibrotide to prevent veno-occlusive disease

By Lisa Kerner

Erie, Pa., Jan. 20 - Gentium SpA said it has begun a phase 2/3 trial with Defibrotide to prevent veno-occlusive disease, a complication of bone marrow and stem cell transplantation, in pediatric patients.

The randomized phase 2/3 study will evaluate the ability of Defibrotide to prevent veno-occlusive disease in 270 pediatric patients undergoing stem cell transplantation at 30 clinical sites in Europe and Israel.

Secondary endpoints are measuring the severity of veno-occlusive disease and the occurrence of transplant-associated microangiopathy in each group.

"Earlier clinical trials of Defibrotide to treat and prevent veno-occlusive disease have shown encouraging results," Laura Ferro, M.D., president and chief executive officer of Gentium, said in the release. "We are pleased to begin this prevention trial in children and hope it will provide evidence of Defibrotide's potential efficacy to prevent veno-occlusive disease."

Defibrotide, a single-stranded DNA, protects the vascular endothelial cells, particularly those of small vessels, from damage and activation. Its beneficial pharmacological effects are due to its anti-thrombotic, anti-inflammatory and anti-ischemic properties, the company said.

Veno-occlusive disease is a blockage of the small veins of the liver leading to liver cell damage. About 20% of patients who undergo stem cell transplantation develop the complication, one-third of which will progress to multiple organ failure, according to a company news release. Nearly 80% of severely affected veno-occlusive disease patients die within three months.

There are no approved therapies to treat or prevent veno-occlusive disease, the company said in the release.

The European Group for Blood and Marrow Transplantation is co-sponsoring the study with additional support from the Deutsche Krebshilfe (German Cancer Aid).

Defibrotide is expected to be the subject of a U.S. phase 3 study as a treatment for severe veno-occlusive disease in the coming weeks. Gentium intends to begin studies of Defibrotide to prevent veno-occlusive disease in the United States by early 2007.

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

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