What is the intent of a Phase 2 clinical trial?

The intent of a Phase 2 clinical trial primarily focuses on determining the effectiveness of a treatment against a specific disease. Following Phase 1, which is primarily concerned with safety, Phase 2 involves a larger group of participants to assess how well the treatment works. This phase is crucial because it provides initial data on whether the treatment has a beneficial effect on the condition being studied, which can then guide further development and testing in subsequent phases.

While identifying harmful side effects and analyzing long-term effects are important considerations in clinical research, these aspects are typically more comprehensively evaluated in Phase 1 and Phase 3 trials, respectively. Phase 2 is not intended to ensure cure efficacy as its objective is focused on effectiveness rather than achieving a cure. Therefore, the specific goal of Phase 2 trials is to assess the impact of the treatment on the disease in a controlled setting to lay the groundwork for larger, conclusive studies.