Italy-based biopharmaceutical company Gentium has reported encouraging preliminary unaudited top-line results from the Phase II/III European pediatric prevention clinical trial of defibrotide.
The Phase II/III European pediatric prevention trial is a multi-center, open label, randomized clinical trial to evaluate the prophylactic use of defibrotide in pediatric patients undergoing stem cell transplantation who are at high risk for hepatic veno-occlusive disease (VOD).
In this two-armed trial, patients were randomly assigned to receive defibrotide. Patients in the prophylaxis arm received 25mg/kg/day of defibrotide in four divided doses beginning at the time of conditioning. Patients in the control arm, however, did not receive defibrotide for VOD prophylactic measures. The primary endpoint of the study was development of VOD within 30 days post stem cell transplantation (SCT) based on the modified Seattle criteria.
The results from this clinical trial demonstrated a 40% reduction in incidence of VOD within 30 days after SCT and achieved a statistical P-value of 0.0539, with a hazard ratio of 1.68 (95% confidence interval of 0.98-2.86), in the intent-to-treat analysis of 180 patients in the prophylaxis arm and 176 patients in the control arm, said Gentium.
In addition, the analysis of data pursuant to the protocol (patients who completed 30 days in the study), which included 164 patients in the prophylaxis arm and 169 patients in the control arm, showed a 40% reduction rate of the incidence of VOD within 30 days and achieved a statistical P-value of 0.0366, with a hazard ratio of 1.78 (95% confidence interval of 1.03-3.08). The data also demonstrated the excellent safety profile of defibrotide showing no difference in adverse events between the prophylaxis and control arms, added Gentium.
Laura Ferro, CEO of Gentium, said: We are encouraged by the preliminary results from the Phase II/III trial of defibrotide to prevent VOD in children and anticipate announcing the final results in the second half of 2009. We look forward to sharing the final results with the European Medicines Agency in order to determine the necessary steps for potential approval.