Hypercholesterolemia, marked by elevated cholesterol levels, is a significant contributor to cardiovascular disease, the leading global cause of death.

Despite advancements in treatment, the complexity of the condition underscores the need for continued innovation in clinical trials to develop effective therapies and refine protocols.

This whitepaper explores the critical aspects of hypercholesterolemia clinical trials, including diagnostic criteria, epidemiology, pivotal endpoints, and the challenges of endpoint determination. It also examines FDA-approved therapies, failed drug candidates, and common pitfalls in trial design, highlighting lessons from past failures. Safety concerns, a key consideration in lipid-lowering therapies, are addressed with strategies for risk mitigation. 

iNGENū’s team of researchers and clinicians is dedicated to advancing hypercholesterolemia  research. Through innovative trial designs and a patient-centered approach, we work to accelerate the development of new treatments that could offer improved options and outcomes for those affected by hypercholesterolemia.