A Study on the Safety and Efficacy of LAE001 in the Treatment of Metastatic Prostate Cancer

Phase I study of this study is a dose-escalating study with enrollment of patients with metastatic castration-resistant prostate cancer who have never received chemotherapy or who have received chemotherapy (chemotherapy failure or intolerance), and preferential enrollment of patients who had failed chemotherapy. The phase II study is a single arm, multicenter study based on ADT, and only patients with metastatic castration-resistant prostate cancer will be enrolled.

According to the results of a completed phase I dose-escalation trial and preclinical data on the drug, the initial dose proposed for this study is 50 mg BID, and one cycle will be 28 days. The escalated doses are 100 mg BID and 125 mg BID, two dose groups. If two or more cases of DLT occur for 100 mg BID, the dose may be reduced to 75 mg BID for investigation. If DLT occurs in two or more out of six patients for a certain dose group, the group with the dose preceding the testing dose will be determined to be the MTD dose group.

RP2D will be determined based on a comprehensive analysis of the safety, pharmacokinetic, pharmacodynamic and efficacy data of dose escalation. Where the MTD is determined, MTD is usually taken as the RP2D, or a dose lower than MTD is selected as the RP2D based on the combined data. If it is determined from the safety data that the dose can still be further increased, but the pharmacokinetic data indicate that the plasma concentration of LAE001 has reached steady-state saturation, the lowest dose that reaches steady-state saturation will be taken as the RP2D. According to the above principles, if the overall incidence of DLT at the dose is \< 1/3, this testing dose will be determined as the RP2D. Six patients will be further enrolled and treated at the RP2D dose for sufficient pharmacokinetic data.

Phase II study is designed as a signle arm, multicenter trial on top of ADT therapy. Its primary objective is to assess the efficacy and safety of LAE001 in patients with metastatic castration-resistant prostate cancer. About 40 patients will be enrolled and assigned to the LAE001 treatment group on top of basic ADT therapy. Participant will be treated until the occurrence of disease progression (clinical evidence required), intolerance, judgement by the investigator that the patient is unsuitable to continue receiving treatment, death, or withdrawal of informed consent.