What are cancer clinical trials?

Cancer clinical trials are research studies with people. They may be done by government agencies, educational organizations, private not-for-profit groups, or commercial businesses. Trials are used to check how safe new cancer tests or treatments are and how well they work.

What are some types of cancer clinical trials?

According to the National Cancer Institute, there are different types of cancer clinical trials. These include:

• Prevention trials. These trials test therapies that might keep cancer from developing in people who have never had cancer. Some of these trials test treatments that might keep a new type of cancer from forming. Others may look at things like activity or diet and cancer risk.

• Early detection or screening trials. These trials look for new ways to find precancers (like colon polyps) or cancer before it causes symptoms, especially in its early stages, when it hasn't spread and is easier to treat.

• Treatment trials. Such trials test new therapies in people who have cancer. Treatment trials often compare the best treatment available today to new and possibly better treatments. They can also test new ways of using existing treatments. Most clinical trials for cancer are treatment trials.

• Supportive care/palliative care trials. These are done to improve the comfort and quality of life for people who have or had cancer, especially with side effects from cancer and its treatment.

• Observational studies. These study people with cancer or a high risk for developing cancer over a long period of time to collect and analyze data. No treatment is given.

What are the phases of a clinical trial?

Most clinical trials go through an orderly series of steps or phases:

• Phase 0 trials. These are the earliest steps in testing new treatments in humans. A small number of people are studied. The goal is to learn how the agent is used by the body and how the agent affects the body. This type of trial does not give information on how safe the agent is or how well the agent treats the cancer. These trials help researchers decide if the treatment should move on to Phase I trial. 

• Phase I trials. These are the first studies to check how a new therapy should be given, how often, and what the highest dosage is that can be tolerated. The focus is to find out information about the safety and side effects of the therapy for the next phase of testing. Phase I trials are done in a small group of people.

• Phase II trials. These provide beginning information about how well the new therapy works. They also give more information about its safety and benefits. A Phase II study usually focuses on a certain type of cancer. It is done in a larger group of people.

• Phase III trials. These compare a promising new therapy, combination of treatments, a procedure and side effects with a current standard of treatment. These trials often have large numbers of people. They may be done at health care providers' offices, clinics, and cancer centers nationwide, or even worldwide.

  • Phase I-III clinical trials are used to make FDA approval decisions about new treatments or existing treatments for new conditions.

• Phase IV trials. These include the ongoing study of the therapy. They look at long-term safety and effectiveness. They take place after the FDA approves the therapy. The therapy is already available for general use.

What is informed consent?

Informed consent is an essential process that explains the important facts about a specific clinical trial. The goal is to protect potential people involved in the study by making sure they understand everything about the trial and the possible treatment. All the details, including the risks and benefits, of the clinical trial will be shared with you before you agree to be in it. You will be told things like:

• What will be studied and how.

• What treatments and medical tests will be used as part of the study.

• What information will be collected.

• Your rights, including your right to leave the study at any time.

• All the treatment choices that might be right for you.

• Who is on the research team and their contact information.

This information may be found in the clinical trial protocol. This is the action plan or guidelines that must be followed during the course of any study. Several research centers may be involved in the study. Each center uses the same protocols to make sure that information from all centers can be combined and compared.

After you learn about all the details and you agree to take part in the study, you'll be asked to sign the consent form. Signing it means that you understand the risks and benefits of the clinical trial plan and agree to take part in the study. Even after signing the form, you may leave the study at any time.

Where are trials conducted?

Trials can be done at large cancer centers, university hospitals, local medical centers, or health care provider' offices. 

Who can take part in a trial?

Each protocol in a trial lists the specific characteristics that participants should have to participate in the study. These characteristics are called eligibility criteria. They are inclusion or exclusion criteria. Criteria that allow you to sign up for a trial are inclusion criteria, such as the type of disease and its stage, as well as the person's age and general health. Criteria that can prevent you from joining a trial are called exclusion criteria.

These criteria help researchers know for sure that the study results answer the research question. They also identify who will benefit in the future from the approach being studied.

How are trial participants protected?

Several procedures are put in place to protect the safety of the participants. Two groups that watch over the safety are the:

• Organization that sponsors the study, such as the National Cancer Institute.

• Institutional Review Board (IRB) that oversees clinical research in a health care organization. The IRB includes health care providers, consumers, patient advocates, and sometimes members of the clergy. The members of the IRB don't have any personal interest in the results of the study. They make sure that the study is done fairly and that there is a low chance of harm to the participants compared to the possible benefits.

A Data Safety and Monitoring Committee is another special group involved in some clinical trials, especially Phase III trials. This group looks at the ongoing test results, keeps track of the safety of the participants, and decides whether the study should go on as originally planned. They can stop the trial early if the main research question is answered sooner than expected or if there are safety concerns.

For information on how to participate in a clinical trial, talk with your provider or visit the National Cancer Institute's clinical trial webpage.

What will your experience be like if you join the trial?

Your study team will provide details on visits, procedures, and guidance on communicating side effects and concerns. You may have more frequent blood tests, imaging tests, or visits to your health care provider than people who are not in a clinical trial. These help the researchers know how the treatment is working and how you are responding to it. Your health care team will want to know if you are having any side effects (adverse events). It's important that you report any symptoms.

Can you leave the clinical trial?

You can stop being part of a clinical trial at any time. Your participation is voluntary. Let your health care team know that you don't want to continue. They may ask why you are leaving. You might want to leave if the trial is causing very bad side effects, not having the impact you wish, or simply because you want to stop.