Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02851407 | Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients | PHASE3 | COMPLETED | 372 | — | — | Sep 1, 2016 | Oct 20, 2020 | Mar 2, 2022 | 114 | United States, Australia +12 |
| NCT00003966 | Defibrotide in Treating Patients With Liver Damage Following Peripheral Stem Cell Transplantation | PHASE2 | COMPLETED | 151 | — | — | Apr 1, 2000 | Apr 1, 2006 | Jan 20, 2017 | 9 | United States |
VOD-free survival is a composite of survival status and VOD occurrence as determined by modified Seattle criteria adjudicated by a blinded independent EPAC. An event is defined as a VOD diagnosis (as assessed by the EPAC) or death, whichever, is earlier, up to and including Day +30 post-HSCT. The values reported below are participants who did not experience VOD or death by Day +30 post-HSCT.
| Arm | Type | Description |
|---|---|---|
| Defibrotide | EXPERIMENTAL | Defibrotide is administered intravenously at a dose of 25 mg/kg/day in addition to best supportive care on the day before the first day of the conditioning regimen and will continue (for those patients without a VOD diagnosis) for a recommended minimum of 21 days and end no later than Day +30 post HSCT |
| Best Supportive Care | OTHER | Best supportive care alone (without the addition of defibrotide) according to institutional guidelines and patient need, is administered on the first day of conditioning and will continue until Day +30 post HSCT or hospital discharge, whichever is sooner, or diagnosis of VOD, if applicable |
| Arm A Lower dose | EXPERIMENTAL | This is a randomized, multicenter study. All patients initially receive the same dose of defibrotide IV over 2 hours every 6 hours on day 1. On day 2, patients are randomized to 1 of 2 doses of defibrotide. \- Arm I: On days 2-14, patients receive a lower dose of defibrotide IV over 2 hours every 6 hours. In both arms, courses repeat every 14 days in the absence of disease progression or unacceptable toxicity |
| Arm B Higher Dose | EXPERIMENTAL | This is a randomized, multicenter study. All patients initially receive the same dose of defibrotide IV over 2 hours every 6 hours on day 1. On day 2, patients are randomized to 1 of 2 doses of defibrotide. \- Arm II: On days 2-14, patients receive a higher dose of defibrotide IV over 2 hours every 6 hours. In both arms, courses repeat every 14 days in the absence of disease progression or unacceptable toxicity |
| Name | Type | Description |
|---|---|---|
| Defibrotide | DRUG | - |
| Best Supportive Care | OTHER | - |
Inclusion Criteria: 1. Patient must be above the age of 1 month as of the start date of study treatment. 2. Patient must be scheduled to undergo allogeneic hematopoietic stem cell transplant (HSCT) (adults or pediatric patients) or autologous HSCT (pediatric patients only) and be at high risk or ve...