Clinical Question: During the hem/onc clinic rotation, on GU Malignancies Clinic day, you see a new patient 28 y/o Caucasian female recently diagnosed with Stg IV Renal Cell Carcinoma –a (local invasion, 2 LN nodes positive, one pulm nodule) who presents for consideration of systemic chemotherapy. Study: TARGET Trial (Treatment approaches in RCC Global Evaluation trial) Escudier et al; Sorafenib in Advanced Clear cell Renal Cell Carcinoma. 2007 Jan 11;356(2):125-34 Study Type: phase multi center randomized double blinded placebo controlled trial. Description of Clinical Trials: Phase I Trial: -assess the safety (pharmacovigilance), tolerability, pharmacokinetics, and pharmacodynamics of a drug. -also normally include dose-ranging, also called dose escalation, studies so that the appropriate dose for therapeutic use can be found. The tested range of doses will usually be a fraction of the dose that causes harm in preclinical models. -Single vs Multiple Ascending Dose Regimens: SAD vs protocols. -usually small studies, N=20-50. -most often include healthy volunteers. -However, there are some circumstances when real patients are used, such as patients who have end-stage disease and lack other treatment options. Usually occurs in oncology and trials of immunomodulatory agents. Phase II Trial: Once the initial safety of the study drug has been confirmed in Phase I trials, Phase II trials are performed to assess efficacy -usually larger pt population N=20-300. -When the development process for a new drug fails, this usually occurs during Phase II trials when the drug is discovered not to work as planned, or to have toxic effects. -Trial Design: Some are designed as case series while some are designed as RCT’s. However, phase II RCT have far fewer patients than the phase trials. -May be subdivided into phase IIA(designed to study dosing requirements) vs Phase IIB(designed to study efficacy) Phase Trial: -randomized controlled multicenter trials on large patient groups. -Large studies: N=300-3000. -aimed at definitive assessment of drug efficacy where exp drug is compared to current standard of care. -most expensive, time-consuming and difficult trials to design and run.
Clinical Question: During the hem/onc clinic rotation, on GU Malignancies Clinic day, you see a new patient –a 28 y/o Caucasian female recently diagnosed with Stg IV Renal Cell Carcinoma (local invasion, 2 LN nodes positive, one pulm nodule) who presents for consideration of systemic chemotherapy. Study: TARGET Trial (Treatment approaches in RCC Global Evaluation trial) Escudier et al; Sorafenib in Advanced Clear cell Renal Cell Carcinoma. 2007 Jan 11;356(2):125-34 Study Type: phase multi center randomized double blinded placebo controlled trial. Description of Clinical Trials: Phase I Trial: -assess the safety (pharmacovigilance), tolerability, pharmacokinetics, and pharmacodynamics of a drug. -also normally include dose-ranging, also called dose escalation, studies so that the appropriate dose for therapeutic use can be found. The tested range of doses will usually be a fraction of the dose that causes harm in preclinical models. -Single vs Multiple Ascending Dose Regimens: SAD vs protocols. -usually small studies, N=20-50. -most often include healthy volunteers. -However, there are some circumstances when real patients are used, such as patients who have end-stage disease and lack other treatment options. Usually occurs in oncology and trials of immunomodulatory agents. Phase II Trial: Once the initial safety of the study drug has been confirmed in Phase I trials, Phase II trials are performed to assess efficacy -usually larger pt population N=20-300. -When the development process for a new drug fails, this usually occurs during Phase II trials when the drug is discovered not to work as planned, or to have toxic effects. -Trial Design: Some are designed as case series while some are designed as RCT’s. However, phase II RCT have far fewer patients than the phase trials. -May be subdivided into phase IIA(designed to study dosing requirements) vs Phase IIB(designed to study efficacy) Phase Trial: -randomized controlled multicenter trials on large patient groups. -Large studies: N=300-3000. -aimed at definitive assessment of drug efficacy where exp drug is compared to current standard of care. -most expensive, time-consuming and difficult trials to design and run.
5.8.09%20Puvvada.%20Sorafenib%20in%20RCC.docx