Patient education: What are clinical trials? (The Basics)

What is a clinical trial? — A clinical trial is a research study that tries to find new ways to improve health. People volunteer to be in clinical trials. Clinical trials can:

●Test new treatments for a certain disease or condition to see if they work better than the current treatments

●Test treatments or lifestyle changes to see if they prevent or lower the chance of getting certain diseases

●Test new ways of finding out if people have certain diseases

In most clinical trials, researchers compare a new treatment with either the "usual" treatment for the disease, or with a "placebo" treatment. A placebo is something that looks the same as medicine and is given the same way, but doesn't have any medicine in it. Not all clinical trials use placebos.

Clinical trials that test new treatments, such as medicines, fall into 1 of 3 types. The type of trial depends on its goal and how much testing the treatment has already had. The different types of treatment trials are:

●Phase 1 (I) trials – In phase I trials, doctors check the safety of a new treatment. They learn how the medicine should be given, how often it should be given, what dose is safe, and what the side effects are.

●Phase 2 (II) trials – In phase II trials, doctors give a new treatment to a group of people with a certain disease. The doctors check the treatment's side effects and how well it works to treat the disease.

●Phase 3 (III) trials – In phase III trials, researchers compare a new treatment with either the usual treatment for the disease or with a placebo. This helps researchers learn if the new treatment is better, worse, or the same as the current treatment or placebo. They also learn if the new treatment has more or fewer side effects than the current treatment.

Who can be in a clinical trial? — It depends on the trial. Some trials (usually phase II or III trials) use only people with certain diseases or conditions, or whose family members have certain diseases or conditions. Other trials use people who are healthy, instead of people with a certain disease or condition.

Not everyone who wants to be in a trial can be in one. The researchers set rules about who can be in each trial. Often, you need to have the disease or health problem being studied. Other rules might have to do with your age and whether you have any other medical conditions. To see if you can be in a trial, you might need to answer questions or have an exam or tests.

What are the benefits of being in a clinical trial? — People can benefit in different ways. People who have the disease being studied can benefit by:

●Having a chance to get a new treatment before it is available to the public

●Having expert doctors treat them and follow their disease closely

●Knowing that they are helping people who will have the same disease in the future

Healthy volunteers can benefit from being in clinical trials, too, by:

●Knowing that they are helping others

●Learning about their health and about research

What are the downsides of being in a clinical trial? — The downsides can include the following:

●The new treatment can have side effects. The side effects might be unknown or unexpected.

●The new treatment might not work as well as the current treatment, or it might not work at all.

●You might not get the new treatment – In general, in studies that compare 2 treatments, half of the people get the new treatment, and the other half get the usual treatment or placebo. Neither you nor your doctor can choose which treatment you get. Also, in many cases, you won't know which treatment you got until the trial is over.

What else should I know about clinical trials? — You should know that:

●It's your decision to be in a trial or not. To help you decide, someone familiar with the trial will talk with you and answer your questions. They will tell you about the trial, the benefits and downsides, and the tests and treatments that you will have. If you agree to be in the trial, you need to sign a consent form.

●Experts check all trials (before and during) to make sure that they are safe.

●You do not have to pay to be in a trial. But your insurance might be billed for some of the treatments or tests that you have during it.

●During the trial, you need to follow all of the instructions about visits, tests, and treatments. If you have any side effects or problems, let the doctor know.

How can I find out about getting in a clinical trial? — To search for clinical trials in the US and worldwide, check the National Institutes of Health website (www.clinicaltrials.gov). If you have a disease, you can also talk with your doctor or nurse. They might know of a clinical trial looking for people with your disease.

If you have cancer, you can search for clinical trials on the National Cancer Institute website (www.cancer.gov/about-cancer/treatment/clinical-trials). Also, the American Society of Clinical Oncology has some videos answering common questions about clinical trials (www.cancer.net/research-and-advocacy/clinical-trials/welcome-pre-act).