Defining Clinical Trials

Defining Clinical Trials

Denise Mouro, R.N., research nurse at the University of Michigan Comprehensive Cancer Center explains the clinical trial recruitment process.

What is clinical research?

Clinical research is research about people. There are several types of cancer-related clinical research studies: Some focus on better ways to prevent or diagnose cancer. Some are designed to improve treatment for people with cancer. Some studies look for new ways to prevent the side effects often associated with existing cancer treatments. Unlike other types of scientific research that take place in a laboratory, clinical research usually takes place in a hospital or clinical setting under the direction of researchers who also are doctors.

What is a clinical trial?

A clinical trial is a specific kind of clinical research study. It's designed to test whether experimental cancer drugs or treatments are safe and effective to use in people. In a clinical trial, an experimental drug or treatment is first given to volunteers to see if the drug is safe and has tolerable side effects, and is then tested against conventional therapies to see which works best in cancer patients.

Kenneth J. Pienta, M.D., explains how important it is for the pool of participants to be diverse.

Where do clinical trial volunteers come from?

People learn about clinical trials in different ways. Sometimes, patients are referred by their primary care doctor or oncologist. Some trial volunteers are cancer patients who want to try experimental drugs or treatments that aren't yet on the market. These patients often find information about a clinical trial on the Internet and may travel long distances to participate. Clinical trials are not available everywhere. Most are located at major medical centers with a national reputation for quality clinical research programs, like the U-M Comprehensive Cancer Center.

Are there enough people volunteering for clinical trials?

There is a nationwide shortage of clinical trial volunteers, especially among racial and ethnic minority groups. Whether you get cancer and how well you respond to treatment is determined, in part, by the genes you inherited from your mother and father. So, it's important to have people with a wide range of genetic backgrounds enrolled in clinical trials, because experimental treatments can work differently in people from different ethnic or racial groups. Certain types of cancer are more common in Black and Hispanic Americans, so it's especially important to have more Black and Hispanic volunteers enrolled in clinical trials.

Denise Mouro, R.N., research nurse at the University of Michigan Comprehensive Cancer Center explains the role of a clinical research nurse when it comes to clinical trials.

Why are clinical trials important?

Without clinical trials, there would be no new treatments for cancer. Under most circumstances, doctors in the United States cannot treat patients with experimental drugs or treatments unless they have been carefully tested and licensed for patient use by the federal Food and Drug Administration. FDA experts analyze the results from clinical trials to determine whether new therapies are safe and more effective than established treatments. Without FDA approval, new cancer drugs or therapies cannot be marketed in the United States.

What are the phases of a clinical trial?

Clinical trials of new cancer drugs or treatments are conducted in a series of stages called phases:

Phase I: researchers find out whether the experimental drug or treatment is safe for people and what side effects it causes.

Phase II: researchers use phase II trials to find out if the experimental drug or treatment is effective against a specific type of cancer.

Phase III: these studies (trials) are designed to compare, in matched groups of hundreds or even thousands of patients, the results from experimental treatment with results from conventional treatment. Unless the experimental treatment is shown to be safe and more effective than conventional treatment, it is unlikely to be approved by the FDA for general use in patients.

Ken Pienta, M.D and Denise Mouro, R.N., discuss side effects, what doctors can do to help and how safety of clinical trial volunteers is protected.

Are clinical trials only for people in late stages of cancer?

Many cancer patients think they can't participate in a clinical trial until conventional treatment stops working. This is unfortunate, because many experimental drugs and treatments work best in the early stages of cancer. Also, many clinical trials will only accept people who haven't received conventional treatments. The best time to volunteer for a clinical trial is as soon as you are diagnosed.

Is it dangerous to be in a clinical trial?

Experimental treatments do have side effects, and some of them can be unpleasant or even dangerous. But conventional treatments can have dangerous side effects, too. Patients enrolled in clinical trials are monitored closely by doctors and research nurses, who watch for early signs of complications and side effects and can stop the treatment immediately if problems develop. Plus, every clinical trial must be approved by an Institutional Review Board or IRB. IRBs include physicians, scientists and community members who review every study in advance to ensure that the benefits to study participants outweigh any risks involved in being part of the study.

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