Clinical Studies

What are clinical trials and studies?

Clinical trials study health and illness in people. They explore new ways to prevent, detect or treat disease.

What is a clinical trial?

A clinical trial is a type of medical research study that helps answer important questions about human health, such as how illnesses and disease are identified, treated and managed. In a clinical trial, researchers test an intervention, like a drug, medical device, procedure or a change in behavior to learn if it is safe and effective in people. Clinical trials depend on volunteers (participants) to answer these questions and improve health.

There are different types of clinical studies; they do not always test medicines. Some look at the long-term effects of treatment or the impact of diet, nutrition and exercise on illness or disease. Some need volunteers with specific illnesses and some need healthy volunteers. Some examine the effects of a new drug, vaccine or treatment and others monitor large populations in normal settings to compare changes over time. 

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How are clinical trials designed and supervised?

Clinical trials are carefully conducted and must follow strict rules and regulations, called a protocol. The protocol is designed to protect the safety of the volunteer to the highest degree. Clinical trials must also be approved by an independent committee, called an Institutional Review Board, or IRB, that examines the risks and benefits of the study and determines if risks are reasonable.


Phases of clinical trials

Clinical trials are conducted as part of one of four phases, and each phase has a specific goal.

Phase I

Phase I trials explore how a new medicine or treatment should be given (orally, topically, intravenously) as well as how often and at what dose. Some Phase I trials add new medicines into known treatment plans. These trials typically include a small group of patients.

Phase II

Phase II trials may include up to several hundred people to further explore the safety and efficacy of a treatment or approach to disease management.

Phase III

Phase III trials may include up to several thousand participants and are focused on gathering more information about the intervention’s safety and effectiveness. After a Phase III trial is successfully completed, the FDA will use this data to determine whether to approve the new medication or medical device.

Phase IV

Phase IV trials evaluate the safety and efficacy of an FDA-approved treatment among a larger number of patients and over a longer period of time. Sometimes, this phase also assesses how well a new treatment works in patients with certain characteristics or in combination with other therapies.

Sources:

  1. National Institutes of Health. NIH Clinical Research Trials and You. Access October 30, 2025. https://www.nih.gov/health-information/nih-clinical-research-trials-you
  2. National Institutes of Health. National Institute on Aging. What Are Clinical Trials and Studies? Accessed October 30, 2025. https://www.nia.nih.gov/health/clinical-trials-and-studies/what-are-clinical-trials-and-studies

Participate in a study

Learn more about the potential benefits of participating in clinical studies.