Clinical Trials

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Clinical Trials

Treatment Overview

What is a clinical trial?

Clinical trials are research studies in which people help doctors and researchers find ways to improve health care. Each study tries to answer scientific questions and to find better ways to prevent, diagnose, or treat disease.

The purpose of a clinical trial is to find out whether a medicine or treatment regimen is safe and effective for the treatment of a specific condition or disease. Clinical trials compare the effectiveness of the study medicine or treatment against standard, accepted treatment, or against a placebo if no standard treatment exists.

What are the phases of a clinical trial?

A medicine or treatment regimen must go through three phases before it is approved for use by the U.S. Food and Drug Administration (FDA).

  • Phase I: A new medicine is tested for the first time on a small group of healthy people or people with specific conditions or diseases. Researchers evaluate the safety of the medicine or treatment, the best dose or schedule to use, and what types of side effects occur. During phase I trials, all of the people involved in the study (patients, doctors, and researchers) know what medicine is being used. These are called nonrandomized, nonblinded studies.
  • Phase II: The medicine or treatment is tested on a larger group of people with specific conditions or diseases. This phase helps researchers find out how well a medicine or treatment regime will work to treat a particular problem. Phase II trials are also usually nonrandomized, nonblinded studies.
  • Phase III: The medicine or treatment regimen is tested on even larger groups. The medicine is studied to find out how well it works compared with standard treatment or placebo and whether it improves specific areas in your life, such as how well you are able to keep up with your usual routine. Most medicines that reach phase III trials will be considered for FDA approval. During phase III trials, participants receive the study medicine, a placebo, or the standard treatment. Neither the participants, the doctors, or the researchers know which person is getting which medicine. These are called randomized and double-blinded studies.
  • Phase IV. Medicines are also studied after they are approved to find new uses for the medicine, different ways to administer it, or additional safety information. For example, a medicine may be studied to see how well it works for a specific population, such as adults over the age of 65 or a certain racial group.
By: Healthwise Staff
Medical Review: E. Gregory Thompson, MD - Internal Medicine
Michael Seth Rabin, MD - Medical Oncology
Last Revised: October 31, 2011

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