The phase III trial will evaluate approximately 300 previously-treated patients with locally advanced and/or metastatic pancreatic cancer who receive best supportive care (BSC) or glufosfamide once every three weeks plus BSC. Best supportive care includes all medical or surgical interventions that a pancreatic cancer patient should receive to palliate the cancer but excludes treatment with systemic therapies intended to kill the cancer cells.
The primary endpoint of this trial is overall survival as measured by time from randomization to death. The timing of the final analysis is therefore event-driven and will be conducted after the 258th death has occurred. In addition, the trial will investigate the potential efficacy of glufosfamide as determined by response rate, duration of response and progression-free survival, as well as safety.
The phase III trial is being conducted under a special protocol assessment (SPA). The SPA agreement with the FDA indicates that if the trial successfully meets its primary efficacy endpoint and satisfies other criteria, the data will provide support for an efficacy claim in a marketing application to the FDA.
The phase II trial, meanwhile, will evaluate up to 28 previously-untreated patients with locally advanced and/or metastatic pancreatic cancer who receive gemcitabine (marketed as Gemzar by Eli Lilly) weekly for three of every four weeks plus glufosfamide administered once every four weeks.
In addition to safety, the trial will investigate the efficacy of glufosfamide in combination with gemcitabine as determined by response rate, duration of response, progression-free survival, overall survival, six- and 12-month survival and change in serum tumor marker levels.