Phase III Clinical Trial: Treatment of Severe Veno-Occlusive Disease with multi-organ failure
The Phase III, historically controlled trial was a multi-center (35 BMT/SCT centres in the US, Canada and Israel) open label study evaluating Defibrotide for the treatment of severe Veno-Occlusive Disease (patients with VOD and multi-organ failure) in hematopoietic stem cell transplant patients. The primary endpoint of the trial was complete response by 100 days following SCT and utilized historical controls (patients screened using medical charts who met the same criteria for VOD with MOF and received the best therapy and supportive care available at the time, but not Defibrotide) as a comparator. Secondary endpoints included survival rate at 100 days and six months post SCT. Patients in the treatment arm received 25 mg/kg/day of Defibrotide in four divided doses.
The historical control database was generated through a sequential, retrospective medical chart review, with final selection of the control group performed by an independent medical review committee (MRC). The MRC remained blinded to patient outcome data throughout the duration of the trial.
Enrolment completed with: 102 patients in the Defibrotide treatment arm and 32 patients selected by the MRC for the historical control arm.
Final clinical trial results for this study were presented at the American Society of Haematology Conference in New Orleans on 5-8 December 2009.