The data safety monitoring board (DSMB) reported that in order for the study to be 80% powered to detect a p-value of 0.01, the necessary statistical hurdle under the current protocol for FDA approval, the sample size should be increased to 160 patients in the treatment arm and 80 patients in the historical control arm.
The DSMB also noted that a sample size of 102 patients in the treatment arm and 51 patients in the historical control arm would be needed to achieve a p-value of 0.05. Furthermore, the DSMB indicated that the data presented thus far do not raise any safety concerns and did not recommend that the trial be stopped for futility.
Gentium has also announced the preliminary results of an independent medical review committee’s (MRC) selection of historical control patients in the trial. After reviewing the available information, the MRC was only able to conclude that 32 out of the 86 patients initially included in the historical control arm definitively met the eligibility criteria and had a confirmed diagnosis of severe veno-occlusive disease (VOD). There are currently 102 patients enrolled in the treatment arm of the study.
Gary Gemignani, CFO of Gentium, said: “We are currently in discussions with our corporate partner regarding the future development of Defibrotide and are also evaluating our strategic options for the company. We continue to make progress in our European Pediatric Phase II/III prevention trial. To date, 345 out of 360 patients have been enrolled and we look forward to reporting results in 2009.”