At this point, approximately 690 patients had been randomized and eligible patients completing the run-in phase were randomized equally between the EVR+Reduced TAC and TAC Control groups.
Because of this logistical difficulty, this trial use an innovative study design where potential patients will be randomized beforehand during a routine clinic visit and not all randomized patients will be enrolled in the phase III trial.
Had the clinical trials been conducted in a non-selected NSCLC patient population, the strong clinical efficacy would likely not have been evident in Phase I/II studies, the therapeutic benefit likewise may not have been seen in a randomized Phase III study, and this compound might never have become a medicine.
To assess the impact of the diagnostic DNA biomarker on patient management, a randomized controlled trial is conducted (in phase IVa), following marketing approval demonstrating that treating doctors' knowledge of the test result improves patient outcome.
The addition of a third cytotoxic agent has been investigated in randomized phase III trials and has not been shown to improve long-term clinical outcomes but is associated with increased toxicity.
Cancer and Leukemia Group B 80303 was a randomized, phase III study in patients with advanced pancreatic cancer treated with gemcitabine plus either bevacizumab or placebo.
The first trial (TOPICAL) was a randomized phase III trial in 670 lung cancer patients which compare erlotinib (n= 350) with placebo (n= 320) [ 28].
The second was a randomized phase II study in which patients with progressive metastatic castrate-resistant prostate cancer received docetaxel with or without imatinib (Mathew et al, 2007).
The first clinical trial demonstrating the clinical activity of lenalidomide in CLL is a randomized phase II study analysing 45 patients with relapsed or refractory disease [ 21].
The ongoing EORTC 24061 study is a randomized phase II study in which cetuximab [chimeric immunoglobulin G1 monoclonal antibody targeting the epidermal growth factor receptor (EFGR)] is combined with TPF for induction.
