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A Guide to Clinical Trials

What is a clinical trial for patients with cancer?

A clinical trial is medical research in a clinic that tests new cancer treatments to see if they can help people with cancer. Before a cancer treatment is studied in people, researchers may test their ideas for years in a laboratory. A clinical trial involving people is one of the last steps in a long and careful process. These studies are done with cancer patients to find new treatments that are safe and effective.

Why are clinical trials important?

Clinical trials are important because they help doctors learn more about cancer and can lead to better treatments and care. Doctors use the results of clinical trials when they decide on a cancer treatment. If a treatment works well in a clinical trial, it may become a new standard treatment that can help many patients.

What are the types of clinical trials?

Clinical trials are done in a series of steps, called phases.

Phase I

These are early studies to find out what amount of the drug (the dose) works the best and how much can be given safely. In Phase I clinical trials, a small dose is given and gradually increased, while doctors watch closely for side effects.

Phase II

A Phase II trial also tests the safety of the treatment and tries to find out whether the treatment is effective. If the treatment doesn't work, no more trials will be done. If it is effective, doctors will plan a Phase III study.

Phase III

The goal of Phase III trials is to find out whether the new treatment is effective. Because Phase III trials are the largest, this is the type of clinical trial that cancer patients usually join.

What is a randomized trial?

A randomized trial is a study in which patients are put into a treatment group by chance. Patients in a randomized trial may or may not receive the drug under study. Randomization helps avoid what is called bias: having a study's results affected by a doctor's beliefs or choices.

What is a double-blind trial?

In a double-blind trial, neither the patient nor the doctor knows which treatment is being given. There is also something called a single-blind trial in which the doctor knows which group the patient is in but the patient doesn't.

What are eligibility criteria?

Every study has a list of rules that decides who can or cannot join a clinical trial. These rules, called “eligibility criteria,” are a list of features a patient must have to join the trial. These may include age, gender (male or female), and type of cancer. Eligibility criteria help doctors know which patients may be helped the most if a treatment works.

Should I take part in a clinical trial?

Once you know you have qualified for a trial, only you can decide whether to join. Before you decide, you should

  • Learn as much as possible about your disease.

  • Find out what clinical trials are open to you by asking your doctor. You can also call or write to the National Cancer Institute (NCI), National Institutes of Health (NIH), or American Cancer Society (ACS).

  • Discuss your feelings about clinical trials with your doctor and/or nurse, family members, and friends to help you decide what is right for you.

What are the benefits and risks of clinical trials?

You can benefit from clinical trials in many ways:

  • Getting new drugs and treatments that are often not available except in clinical trials.

  • Getting regular and careful medical care from a team that includes doctors and nurses.

  • Being carefully watched for any side effects.

  • Having a more active role in your own healthcare.

  • Being among the first to be helped if the treatment being studied is found to work.

  • Helping to find new treatments that may improve cancer patients' health in the future.

Clinical trials may also have some risks:

  • New treatments may have side effects that doctors don't know about. This is especially true in early phases of clinical trials.

  • New treatments may not work at all or as well as current treatments.

  • Even if a new treatment helps others, it may not work for you.

  • Those in “randomized trials” will not be able to choose the treatment they get.

  • Health insurance may not pay for the study.

  • You may have to make more visits to the doctor than you would if you weren't in a clinical trial.

Where do clinical trials take place?

Clinical trials take place all over the country. If you were in a clinical trial, you might get your treatment at a cancer center, a university hospital, a veterans or military hospital, a local medical center, or your physician's office.

What happens during a trial?

If you decide to be in a clinical trial, you will work with a research team. Team members may include doctors, nurses, social workers, and dieticians. They will provide your care, watch you closely, and give you exact instructions about the study. Being in a clinical trial may mean that you will have more tests and doctor visits than if you weren't in the study. Team members also may continue to stay in touch with you after the trial ends. It is important to follow the research team's instructions.

Could I be given a placebo?

A placebo, also called a “sugar pill,” contains sugar or salt but looks exactly like the drug being used in some studies. Studies with people who have cancer rarely use placebos.

What happens when a clinical trial is over?

After a Phase I or II trial is completed, researchers look carefully at the results and decide whether to move on to the next phase of clinical trial or stop studying the treatment because it is not safe or effective.

When a Phase III trial ends, researchers look at the results and decide whether or not to submit for FDA approval. The researchers will then tell the medical community and the public of the study results regardless of the outcome.

If a new approach is proven safe and effective in a clinical trial the drug may be approved by the FDA.

What are my rights in a clinical trial?

  • Before and during a clinical trial, you have a number of rights. Knowing these rights can help you make informed decisions.

  • Being in a clinical trial is up to you. Talk with your doctor. Together, you can make the best choice for you.

  • If you decide to join a clinical trial, doctors and nurses will watch very carefully how the treatment affects you throughout the entire study.

  • If you experience a serious side effect, you may be taken off the study.

  • You have the right to leave a study at any time.

What is informed consent?

Informed consent is one of the most important rights someone in a clinical trial has. You must be told all the facts about a study before you decide whether to join. There will also be a written consent form explaining the study that you can take home to read and discuss. The consent form will include information about

  • How the study works.
  • The treatments given in the trial.
  • Possible risks and benefits.
  • Tests you may be given.

People who agree to be in the study are asked to sign the informed consent form.

Who can answer my questions about clinical trials?

People with cancer and their families have a lot of questions when they are thinking about joining a clinical trial. It is important to discuss all of your questions with your doctor, a cancer specialist (oncologist), and the clinical trial team. Always remember that when it comes to your medical care, no question is silly.

Who pays for my treatment in a clinical trial?

Even if you have health insurance, your insurance may not pay all of the costs for a clinical trial. This is because some health plans think of clinical trials as “experimental” or “investigational.” In many cases, it can help to have someone from the study team call your health plan and talk to them directly about what will be paid.

Many states require that health plans pay for certain clinical trials. You can find more information about state laws on the Internet. One site with this kind of information is the NCI's State Initiatives and Legislation Digest Page.

How can I find a clinical trial for my kind of cancer?

If you are interested in joining a clinical trial, talk to your doctor. You can also contact cancer organizations that help people find information about clinical trials in their area.

The National Cancer Institute (NCI) is the lead agency for cancer research in the United States. The NCI has a web site that allows people to search for information about clinical trials by type of cancer, location of study, and type of treatment.

The National Institutes of Health (NIH) has a web site that provides regularly updated information about federally and privately supported clinical trials.

The American Cancer Society (ACS) provides a Clinical Trial Matching and Referral Service database containing more than 3,000 clinical trials.

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