What is a Clinical Trial?

In cancer research, a clinical trail is a study conducted with cancer patients, usually to evaluate a new treatment. Each study is designed to answer scientific questions and to find new and better ways to help cancer patients.

The search for good cancer treatments begins with basic research in laboratory and animal studies. The best results of that research are tried in patient studies, hopefully leading to findings that may help many people.

Before a new treatment is tried with patients, it is carefully studied in the laboratory. This research points out the new methods most likely to succeed, and, as much as possible, shows how to use them safely and effectively. But this early research cannot predict exactly how a new treatment will work with patients.

With any new treatment there may be risks as well as possible benefits. There may also be some risks that are not yet known. Clinical trails help us find out if a promising new treatment is safe and effective for patients. During a trial, more and more information is gained about a new treatment, its risk, and how well it may or may not work.

Standard treatments-the ones now being used-are often the base for building new, hopefully better treatments. Many new treatments are designed on the basic of what has worked in the past, in efforts to improve on this.

Only patients who wish to, take part in a clinical trial. You may be interested in or asked to enter a trial. Learn as much as you can about the trial, before you make up your mind.

Why are clinical trials important?
Advances in medicine and science are the results of new ideas and approaches developed through research. New cancer treatments must prove to be safe and effective in scientific studies with a certain number of patients before they can be made widely available.

Through clinical trials, researchers learn which approaches are more effective than others. This is the best way to test a new treatment. A number of standard treatments were first shown to be effective in clinical trials. These trials help us new and better treatments.

Why would a patient be interested in a clinical trial?
Patients take part in clinical trials for many reasons. Usually they hope for benefits for themselves. They may hope for a cure of disease, a longer time to live, or a way to feel better. Often they want to contribute to a research effort that may help others.

Based on what researchers learn from laboratory studies, and sometimes earlier clinical studies and standard treatments as well, they design a trial to see if a new treatment will improve on current treatments. The hope is that it will. Often researchers use standard treatments as the building blocks to try to design better treatments.

Although there is always a chance that a new treatment will be disappointing, the researchers involved in a study have reason to believe that it will be as good as, or better than, current treatments.

The patients in a clinical trial are among the first to receive new research treatments before they are widely available. How a treatment will work for a patient in a trial can’t be known ahead of time. Even standard treatments, although effective in many patients, do not carry sure benefits for everyone. But, patients should choose if they want to take part in a study only after they understand both the possible risks and benefits.

The patients who take part in clinical trial procedures that do prove to be better treatments have the first chance to benefit from them. All patients in clinical trials are carefully monitored during a trial and followed up afterward. They become part of a network of clinical trials carried out around the country. In this network, doctors and researchers pool their ideas and experience to design and monitor clinical studies. They share their knowledge from many specialties about cancer treatment and care. Patients in these studies receive the benefit of their expertise. At cancer centers, patients receive care from a special research team. Through new programs, community hospitals and doctors are also coming more and more into the research network.

Are there risks or side effects in clinical trials?
Yes. The treatments used in clinical trials can cause side effects and other health risks depending on the type of treatment and the patient’s condition. Side effects vary from patient to patient.

Because clinical trials are research into new areas of treatment, the risks involved are not always known ahead of time, though efforts have been made to find out what they might be. For this reason, trials can carry unknown dangers and side effects as well hoped-for benefits.

Patients need to know what is involved in a study-what side effects may be expected-and, as much as possible, what unknowns or uncertainties they may be facing. Your doctor or nurse will tell you about the treatments being tested and will give you a form to read that discusses the risks and hoped-for benefits. If you agree to take part, you will be asked to sign a form, called the informed consent form. Before you sign, be sure you understand what risks you face. Ask the doctor or nurse to explain any parts of the form or the trial that are not clear. If you do not want to be in the trial, you may refuse. Even if you sign the form, you are free to leave the trial at any time and can receive other available care.

In clinical trials, most side effects are temporary and will gradually go away once treatment is stopped. For example, some anticancer drugs cause hair loss and nausea and some do not. They can also effect the bone marrow, which produces blood cells. During treatment, the number of blood cells, called blood counts, may fall too low. Since this could lead to possible infection or other problems, patients have their blood counts checked often, Luckily, bone marrow has a great ability to replace blood cells, so that blood counts can usually return to normal.

Some side effects in clinical trials can be permanent and serious, even life-threatening. Also, certain side effects may not appear until later, after the treatment is over. (These “late” effects may include damage to a major organ like the heart, lungs, or kidneys; sterility; or a second cancer.) Many cancer patients are now living longer, largely because of better treatments. Researchers are concerned and are trying to prevent late complications of treatments.

As a patient, it can be hard to decide about your treatment. There are a number of things to consider. Cancer is a life-threatening disease that causes symptoms of its own that are not related to treatment. In each case, the unavoidable risks of the cancer itself, and your condition, should be weighed against the potential risks and benefits of a new research treatment. Standard treatments, as well as treatments in clinical trials, can also cause side effects and risks.

Why does cancer treatment have side effects?
Any medical treatment can carry the potential for side effects in some patients. Cancer treatment is particularly powerful, because it is designed to destroy constantly dividing cancer cells. It can also affect healthy dividing cells and this can cause side effects. The challenge to researchers has been to develop treatments that destroy cancer cells but do not harm healthy cells.
What is being done to lessen side effects of treatment?
Cancer researchers are trying to make cancer treatment more effective and lessen its side effects for the cancer patient. Results of such efforts include:

• New anticancer drugs with less side effects

• Better anti-nausea medicine

• Some shorter periods of time on anticancer drugs

• Special ways to protect normal tissues during radiation therapy

• New methods of surgery that are less extensive and less damaging to the body

• Psychological support programs and information on ways to cope during difficult times (How patients feel during and after treatment is important)

Questions to ask if you are thinking of entering a clinical trial?
Are you eligible for a clinical trial?
Every clinical trial is designed to answer a set of research questions. If you fit the guidelines for a trial, you may be eligible to take part. Each study enrolls patients with certain types and stages of cancer and certain health status. A study that involves two or more treatments can yield reliable answers only if all the patient cases are the same so they can be compared with one another.

Before you and your physician make a decision about your treatment (whether it is in a clinical trial or not), your type of cancer will be diagnosed and “staged.” Staging tells how far the disease has spread. Deciding on treatment depends on many things, including the stage of the disease and your general health. You would most likely be referred to a trial by your own doctor or by a doctor who knows your case. Some patients find out about trials from other sources. In any case, you must have a reasonable understanding of your role in a research study and be freely willing to take part in it. Ask what you can expect if you take part in a trial.

What trials are available for your type of cancer?
There are many ways to find out what your treatment choices are. Talk with your doctors and get the opinion of cancer specialists (oncologist). You should not be afraid to ask for a second opinion. A helpful treatment information system called Physicians Data Query (PDQ) is supported by the National Cancer Institute. PDQ can give your doctor the latest information on clinical trials being offered around the country for each type and stage of cancer. This ready reference is kept up to date. Your doctor can check it from a medical library or personal computer.

The Cancer Information Service (CIS) is another source of information. This program, also sponsored by the National Cancer Institute, answers cancer-related questions from the public, cancer patients and their families, and health professionals. If you have questions, call the toll-free number: 1-800-4-CANCER and you will be connected to the CIS office serving your area. Spanish-speaking CIS staff are also available.

What is best for you?
This is a big question. Finding answers and making decisions are often hard for a cancer patient. The diagnosis of cancer and deciding what to do about it can be overwhelming, and you may be confused and upset. It is important to discuss your options with medical experts- including your own doctor—and with those close to you. Your personal doctor, who may be your family doctor, and cancer specialists can counsel you about your choices for standard treatment or clinical trials.

Talk to them and ask questions about the problems you are facing. If you understand what is going on, you can help your doctor work with you more effectively. You may want to take a friend or relative along with you when you talk to your doctor about your case.

Take time to ask your questions and to discuss what you want to know. It may help you and your doctor if you plan what to ask and write questions down ahead of time. No question is foolish. Learn what is available to you. Find out your choices and the risks and benefits of each.

Each patient is different. You are an individual with individual needs, and your health is important. If you are a parent of a child with cancer, of course, you have great concerns about making the best decision for your child’s care.

As you decide about treatment, if it is in a clinical trial or not, remember that you are not alone. There are many people to help you-doctors, nurses, social workers, clergy, your family, friends, and other patients. Although it is YOUR decision, they can help you think about it and decide what is best for you.

What are important questions to ask about a clinical trial?
If you are thinking about taking part in a clinical trial, here are some important questions to ask:

• What is the purpose of the study?
• What does the study involve? What kinds of tests and treatments? (Find out what is done and how it is done.)
• What is likely to happen in my case with, or without, this new research treatment? (What may the cancer do and what may this treatment do?)
• What are other choices and their advantages and disadvantages? (Are there standard treatments for my case and how does the study compare with them?)
• How does the study affect my daily life?
• What side effects could I expect from the study? (There can also be side effects from standard treatments and from the disease itself.)
• How long will the study last? (Will it require an extra time commitment on my part?)
• Will I have to be hospitalized? If so, how often and for how long?
• Will I have any costs? Will any of the treatment be free?
• If I am harmed as a result of the research, what treatment would I be entitled to?
• What type of long-term follow-up care is part of the study?

What is informed consent?
Informed consent, a key part of a good trial, is required in studies that are federally regulated or funded as well as by many state laws. Informed consent means that, as a patient, you are given information so you can understand what is involved in a trial, including its potential benefits and risks, and then decide freely to take part in it or not. The nature of the treatment is explained by the doctors and nurses in the trial. You are given an informed consent form to read and consider carefully. Ask any questions you may have. Then, if you agree to take part, you can sign the form. Of course, you can also refuse.

The informed consent process is an ongoing process. If you enter a trial, you will continue to receive any new information about your treatment that may affect your willingness to stay in the trial. Signing a consent form does not bind you to the study. You can still choose to leave the study at any time.

What is it like to be a patient on a clinical trial?
Whether cancer patients are in research study or not, they face a new world of medical terms and procedures. For some people, myths and fears of “experimentation” or of being a “guinea pig” come with the idea of clinical trials. And, surely, there are fears of the unknown. Understanding what is involved can ease some of your anxieties. Patients in a clinical trial, for example, receive their care in the same places that standard treatments are given-at cancer centers, hospitals, clinics, or doctors’ offices.

Because a growing number of cancer specialists are now in private practice in the community, most cancer care can be given in an area near your home. Doctors, nurses, social workers, and other health professionals from many different specialties may help care for you. They are working together for your good. There is consideration for your privacy and well-being.

If you join a research study, you will be watched closely and data on your case will be carefully recorded. You may receive more examinations and tests than are usually given. (These are to follow your progress as well as to collect study data.) Of course, tests can carry certain risks and benefits or discomforts of their own. Although they can be inconvenient, these tests can ensure an extra ounce of observation along the way.

During the course of a study, if it is clear that a treatment is not in your best interest, you will be removed from the study and you can discuss other options with your physician.

Can you leave a trial at any time?
Yes, Just as you can refuse to join a study, you may leave a study at any time. Your rights as an individual do not change because you are a patient in a clinical trial. You may choose to take part or not, and you can always change your mind later, even after you enter a trial.

You may also refuse to take part in any aspect of the research. If you have questions at any time about any part of the study, be sure to ask your doctors. If you are not satisfied with the answers, you may consider leaving the study. If you decide to leave, it will not be held against you. You can freely discuss other possible treatments and care with your doctors and nurses.

Source: What Are Clinical Trials All About? A Booklet for Patients with Caner, NIH Publication#92-2706, U.S. Department of Health and Human Services, Public Health Service, National Institutes of Health, National Cancer Institute, June 1992.

Robin Lentz, Certified Clinical Research Associate - 530-225-7471