Gentium starts Ph II/III Defibrotide study

Italian biopharmaceutical firm Gentium SpA says it has begun a Phase II/III trial of its drug Defibrotide, a single stranded DNA molecule, in the prevention of veno-occlusive disease, a complication of bone marrow and stem cell transplantation in pediatric patients.

The study, which will enroll 270 children who are currently undergoing stem cell transplantation, at 30 clinical sites in Europe and Israel, will assess the compound in terms of its safety and efficacy in the treatment of VOD. The program is being co-sponsored by the Dutch European Group for Blood and Bone Marrow Transplantation, and the German charity Cancer Aid, and will be led by Selim Corbacioglu from the Department of Pediatrics at the University of Ulm, Germany.

Laura Ferro, president of Gentium, commented: "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. Dr Ferro added that the compound is likely to be the subject of a Phase III trial in the USA as a treatment for severe VOD in the next few weeks.