In Phase II trials, the investigational medicinal product is tested for efficacy (and safety). The studies conducted are typically therapeutic exploratory studies which try to discover whether the medicine treats the intended disease or condition.
Early Clinical Development refers generally to the first studies of a medicine in humans – typically known as Phase I and Phase II trials. Studies in early clinical development focus on the safety and tolerability of the new medicine. They also try to show that the medicine can have the intended effect.
The four phases of clinical development are explained in terms of objectives and features.
Phase II clinical studies, or proof of concept studies, begin to test the effect of a medicine on the target disease in humans. These studies are usually run as double-blinded, randomised studies in multiple sites.