Statistical Analysis Plan. This trial uses a Simon two-step design. In step 1, we will evaluate the overall response rate to possibly stop for futility according to the plans described in the sample size determination. In addition, after step 1 we will also evaluate the dose of AE37. If 4 or more of the first 13 patients (> 25%) have Grade 3 or higher toxicity we will de-escalate the dose from 1000 to 500 micrograms daily. Following step 2, we will declare the combination therapy suitable for further study if at least 8 of 29 total patients have objective response. 95% confidence intervals will be reported along with the objective response rate. All patients with a baseline tumor measurement and receiving any protocol treatment will be included in efficacy analysis. All secondary aims will be analyzed after the end of the study, that is after step 1 if the study closes early or after step 2 otherwise. ▇▇▇▇▇▇-▇▇▇▇▇ estimates of PFS and OS will be provided. Tabulations of maximum Grade observed for each patient for each observed toxicity will be provided along with a summary of maximum Grade observed for each patient for any toxicity.
Appears in 2 contracts
Sources: Clinical Trial Collaboration and Supply Agreement (Nugenerex Immuno-Oncology, Inc.), Clinical Trial Collaboration and Supply Agreement (Generex Biotechnology Corp)