Clinical Trial Scenario: OncoZap for Pancreatic Cancer
A pharmaceutical company has developed a novel targeted therapy, "OncoZap," for a rare, aggressive form of pancreatic cancer that has shown promising anti-tumor activity in preclinical in vitro and in vivo models. There are no currently approved targeted therapies for this specific subtype of pancreatic cancer. The company plans to initiate the first-in-human study of OncoZap. The primary objectives are to evaluate the safety and tolerability of increasing doses of OncoZap in a small group of patients with advanced pancreatic cancer, to determine the maximum tolerated dose (MTD), and to characterize its pharmacokinetic profile. The study will enroll approximately 20-30 patients who have exhausted standard treatment options. A secondary objective is to gather preliminary data on anti-tumor activity.
Based on the scenario described, what is the most appropriate clinical trial phase for the planned study of OncoZap? Justify your choice by explaining the primary objectives and characteristics of this phase that align with the scenario.
In the context of clinical trials, clearly define 'primary endpoint' and 'secondary endpoint', highlighting their distinct purposes. Consider a clinical trial investigating a new oral anticoagulant, 'ThromboClear,' for the prevention of stroke in patients with atrial fibrillation. For this trial, propose one plausible primary endpoint and two plausible secondary endpoints. For each endpoint, explain your rationale for its selection.
Explain what randomization is in the context of clinical trials, including its fundamental purpose. Discuss in detail the critical importance and key benefits of randomization in ensuring the internal validity and reliability of study results. Specifically address how it minimizes selection bias and ensures comparability between treatment groups.
Explain the concept of blinding in clinical trials, differentiating between single-blind, double-blind, and triple-blind studies. Discuss the critical importance of blinding in minimizing various forms of bias (e.g., observer bias, participant bias, placebo effect) and maintaining the integrity and objectivity of clinical trial data. Provide concrete examples of how bias could arise and affect trial outcomes without proper blinding.