Clinical Research Trials

A clinical research study, also called a clinical trial, is a carefully designed and closely regulated study of the safety and effectiveness of new, innovative procedures, medications or devices. The purpose of any study is to determine if a new therapy will be effective and safe in preventing, diagnosing or treating people with specific diseases or conditions.

Clinical research can be a long and detailed process, beginning in the laboratory where proven theories are tested. If successful, the next step is the preclinical phase.

Preclinical Research

This is done before clinical trials (testing in humans) can begin. The main goal of preclinical research is to determine if a product is safe. This applies to drugs, devices and gene therapy. Each type of product may undergo different types of testing. The information collected from these studies is vital so that safe human testing can begin. Drug testing may involve animal testing in two species.

Clinical Research Phases

Clinical research is made up of four phases:

• Phase I research studies are the first step in testing any new treatment in humans. These studies usually have 20 participants or less and are designed to show whether a treatment is safe in humans. Often this includes healthy volunteers, and in drug studies can include increases in dose to evaluate the best dose.
• Phase II trials are further tests of safety in a larger number of people, usually 50-200. In addition to safety, effectiveness is beginning to be evaluated, and in drug trials, final dosing regimens are often determined at this phase.
• Phase III trials are done to prove the effectiveness of the new treatment. Most of these studies compare the new treatment to current standard therapy. In drug studies, this is done by comparing the new study drug to a placebo. A placebo is a harmless, inactive substance. One group of patients will receive the active study drug and one group will receive placebo, so that it can be determined whether the study drug is worse, equal or better than current standard therapy. To prevent any bias on the part of the patient or researchers, the study is ‘blinded’. This means that the patient, the research staff and the doctor do not know which group a patient is in. When the study is complete, data is analyzed and submitted to the FDA for approval of the new treatment.
• Phase IV trials are done to determine whether a drug already approved by the FDA could be used for new treatments. For example, a drug that was was approved for prevention of clots in orthopedic patients, then being studied in patients with heart disease who need clot prevention.