An open-labeled, randomized, multicentered, phase IIa study for advanced cancer treatment by gambogic acid injection (THS).

e13095 Background: Gamboge acid is a pure active compound isolated from the Camboge (Garcinia Morella Desv.), a traditional Chinese herb medicine. Based on preliminary results of the completed phase I study, this phase IIa study intends to compare the efficacy and safety of different dosage schedule. Methods: The patients according to enrollment criteria were randomly signed into two arms. Arm A: a daily intravenous infusion of gamboge acid injection (THS) 45mg/m2 for 5 days for every two weeks with 21 patients. Arm B: THS 45mg/m2 intravenous infused every other day for 5 times, per every couple of weeks with 26 patients. All patients in both arms have received two consecutive courses of treatment prior the safety and efficacy evaluation. Results: Observed an objective remission rate (ORR) of 14.29% and an overall disease control rate (DCR) of 76.2% in arm A compare to observed an ORR of 0.00% and a DCR of 61.5% in arm B. Except ORR with an insufficient number of zero, DCR comparison was statistically significant (p=0.0456) with a positive RR and OR and its 95% confidence intervals. The observed adverse events or reactions mostly were grade I and II rather than grade III and IV, and those adverse events or reactions were observed in most cases after patients have received the trial drugs. There was no statistically significant difference in incidence rate of adverse events or reactions between the two armed groups. Conclusions: The preliminary results of the exploratory study indicated favorable safety profiles at 45mg/m2 in this phase IIa clinical study. The 76.19% DCR observed in Arm A is superior to that in Arm B. There was no statistically significant difference in incidence rate of adverse events or reactions between the two arms.