What is a clinical trial? A clinical trial is a research study designed to test potential disease treatments that have already shown promise in the laboratory or in a limited number of patients through a small pilot study. The goal of a clinical trial is to determine if the treatment works with human patients and if it is safe.   Clinical trials are often sponsored by the federal government (through the National Institutes of Health and other agencies), by private industry (pharmaceutical and biotechnology companies), and by medical institutions, foundations and other groups. For a list of current clinical trials, click here.

How does a clinical trial work?
Before a trial can begin, the U.S. Food and Drug Administration (FDA) must decide whether to grant approval to test a new drug or treatment approach on human subjects. The FDA bases its decision on data collected from extensive laboratory and preclinical research, which may include animal subjects and experiments using human tissue samples. The data is submitted as part of an Investigational New Drug application (IND). With FDA approval of this application, a clinical trial can begin. 

Phase I evaluates the safety of the treatment on a small number of participants (usually 20-80 healthy people), establishes the correct dosage, and identifies side effects. The experimental treatment must pass the Phase I safety test before the study can proceed to the next phase.

Phase II tests the effectiveness of the experimental drug or treatment on a larger group (100-300 patients). Does the drug work the way it is intended to? Phase II can last for several months to two years.

This phase is often a randomized trial, which means that one group of patients will receive the experimental drug while the other group—the control group—receives  a placebo (a harmless substance with no active ingredients). The trial may also be a double blind trial, meaning that neither the participants nor physician or researcher administering the drug and monitoring the patients know who is getting the experimental drug and who is getting the placebo.

Phase III provides more information on the benefits and drawbacks of the treatment, which is tested on a much larger group of people (several hundred to several thousand) and can last for several years. Phase III is usually a randomized, double blind, placebo controlled trial comparing the experimental treatment to already established treatments to see which is most effective and safe, and has fewer side effects.

Once Phase III is successfully completed, the pharmaceutical company will request FDA approval for putting the drug on the market and making it available to patients.

Phase IV takes place after the drug has been approved by the FDA, and is not always required. This phase may be conducted to follow up on long-term effectiveness and side effects of the drug, and its impact on the patient’s quality of life. Sometimes a pharmaceutical company may choose to conduct a Phase IV trial to compare its drug to competing products. 

Benefits to participating:
As a patient participating in a clinical trial, you can gain access to a promising drug in Phases II and III of the study, before it is available in the marketplace; play an active role in your own health care; receive excellent care during the trial from a health care team; and benefit other patients by contributing to research.

Risks to participating: 
You may experience side effects from the experimental treatment that could range from unpleasant to life-threatening; the treatment may not be effective; the protocol may be time-consuming and demanding, requiring more doctor visits, sometimes complicated dosages and procedures and more detailed monitoring.

Who can participate:
Potential study participants must meet certain criteria to qualify. These requirements are called inclusion and exclusion criteria and are based on such considerations as age, gender, the type and progress of the disease, other medical conditions and more. These criteria are used to reduce risks to the participants while meeting the requirements of the research plan.

Protecting the safety of participants:
Every clinical trial must first gain approval to go forward from an Institutional Review Board (IRB), which is usually comprised of physicians and lay people.  The IRB reviews the trial protocol—the study plan—to insure that it does not present unnecessary risk to the participants and that the participants’ rights are protected. Clinical trials are subject to the same ethical and legal codes as medical practices, and most trials are federally regulated.

A team of health care professionals—doctors, nurses, sometimes a social worker and other health care experts—works with the participants throughout the trial. The team checks the subjects’ health at the beginning and throughout the trial, provides information and instructions about the protocol, and administers and monitors the treatment during the trial. Participants usually have more frequent contact with the health care team than they might normally have with their physician. The health care is usually free and may be conducted from doctor’s offices, clinics, hospitals, or universities.

Participants can leave a clinical trial at any time and are simply encouraged to advise the health care team if they choose to leave the trial.

Making the decision to participate:
Because there can be serious risks involved in participating in a clinical trial, the decision should be made only after thorough discussions with the research team, your own health care team and your loved ones. It’s also important to remember that not everyone can qualify for a given trial.

Asking questions and gathering information will help you in your decision making. Questions you might consider asking are:

• What is the purpose of the trial?
• What tests and treatments will be used?
• What is known so far about the experimental treatment?
• What are the benefits, risks and side effects compared to your current treatment?
• How will your safety be protected?
• How will the trial affect your daily life?
• How long will the trial take?
• Who will conduct the trial and what are their credentials?
• Who is funding/sponsoring the trial?
• Will you receive payment for participating and/or be reimbursed for expenses?

Informed Consent: 
People participating in a clinical trial are required to sign an Informed Consent form which explains the details of the trial including: the experimental treatment;  possible risks and benefits, tests that may be given, as well as information on treatments already available for your condition.