Jazz Pharmaceuticals plc announced that the first patient has been enrolled in a Phase 3 clinical trial comparing the efficacy and safety of defibrotide versus best supportive care (BSC) in the prevention of hepatic veno-occlusive disease (VOD) in adult and pediatric patients undergoing hematopoietic stem cell transplant (HSCT) who are at high risk or at very high risk of developing VOD. The defibrotide clinical trial will be conducted across approximately 100 medical centers in the United States (U.S.), Canada, Asia Pacific and countries in the European Union (EU).
"The initiation of this trial is another step forward in Jazz's development program for defibrotide as a treatment option for patients who are at high risk or very high risk of developing this life-threatening condition," said Karen Smith, M.D., Ph.D., global head of research and development and chief medical officer of Jazz Pharmaceuticals. "If the data from this trial are positive, they may be used to pursue regulatory approval of a label expansion for defibrotide in both the U.S. and EU to include the prevention of hepatic VOD in high risk patients following HSCT."
The Phase 3 trial is a randomized, open-label, multi-center trial with an adaptive design comparing the efficacy of defibrotide vs. BSC in the prevention of hepatic VOD. The trial is expected to enroll approximately 400 adult and pediatric patients undergoing HSCT who are at high risk or very high risk of developing VOD. High (or very high) risk patients are identified due to the clinical regimen required to prepare them for HSCT, as well as their prior medical history and concomitant disease.
The adaptive design allows for an interim analysis with pre-defined stopping points for early success or failure as well as the option to increase enrollment if needed to preserve the statistical power of the trial.
For patients randomized to receive defibrotide prophylaxis, defibrotide will be administered starting on the day before the first day of the conditioning regimen for a recommended minimum of 21 days and ending no later than Day +30 post HSCT. The primary endpoint is VOD-free survival at Day +30 post-HSCT
