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Colorectal Cancer Resources & Education

What You Need to Know About Clinical Trials

A Guide for People With Cancer

Developed by Pfizer in partnership with the Colon Cancer Alliance

Why You Need to Know About Clinical Trials

As a person with cancer, or a loved one of that person, it’s important to be well informed. You need to know about all your treatment options. This includes treatments that may be offered through clinical trials.

A clinical trial may offer you a new treatment option. Whether you decide to take part in one is up to you. This booklet is all about clinical trials. It will help you decide if a clinical trial is right for you.

What Are Clinical Trials?

Clinical trials are research studies. They are done with volunteers. They look for better ways to prevent, screen for, or treat a disease. Cancer treatment clinical trials test new:

Medicines (chemotherapy and other drugs)
Approaches to surgery or radiation therapy
Combinations of treatments
Methods of treating cancer, such as gene therapy

A treatment is tested before a clinical trial starts. The tests are done in labs or on animals. They check to see how safe and effective the treatment is. The new treatment must be as good as or better than standard ones. The standard treatments are the best ones available to people.

Why Are Clinical Trials Important?

Clinical trials are important. Cancer treatment clinical trials:

Contribute to progress in the understanding of and fight against cancer
Answer important scientific questions and lead the
Determine if a particular treatment is safe and effective
May help the people who take part extend or improve their lives

How Are Clinical Trials Conducted?

Strict scientific and moral standards guide clinical trials. These standards are designed to:

Produce credible research
Protect the rights and well-being of participants

To ensure these goals are met, every clinical trial has:

1. A protocol (PRO-tuh-call), or action plan, that outlines:

What will be studied
Why and how it will be studied
Who will take part and how they will be chosen
How results of the trial will be evaluated

The trial must always use its protocol. Many groups sponsor (fund) clinical trials. They approve the protocol, too. Trial sponsors include:

The National Cancer Institute (NCI)
Pharmaceutical and biotechnology companies
Medical institutions (such as hospitals or clinics)
Individual doctors
Partnerships between universities, government agencies, or doctors and pharmaceutical or biotechnology companies
Private or government organizations

An Institutional Review Board (IRB) also approves the protocol and:

Includes health, clergy, and community members
Makes sure the trial is fair and safe

2. Eligibility criteria, or guidelines, which often include:

Age
Sex
Type and stage of cancer

Medical history
Current health status

Each trial has its own set of guidelines. They say who can be in a clinical trial.

3. Phases of research

Most clinical trials go through a series of steps called phases that include:

PHASE 1 TRIALS

During this phase, the new treatment is tested to find out:
- Whether the treatment is safe—and if so, the amount (dose) that is safe to take
- How it should be given (eg, by mouth or injection) and how many times a day
- Whether the treatment has any harmful side effects
Include only a small number of people not helped by other treatments
Usually take place at only a few locations

PHASE 2 TRIALS

Test how well the new treatment works (eg, if blood test results improve)
Usually include fewer than 100 participants

PHASE 3 TRIALS

Compare results (eg, better survival rates, fewer side effects) of people on new treatment with people on standard treatment
May include hundreds or even thousands of people
Take place all across the country

In Phase 3, researchers assign you to the new or the standard treatment by chance. If you get the new one, you’re in the treatment group. If you get the standard treatment, you’re in the control group.

This process is called randomization (ran-duh-mih-ZAY-shun). It is done to avoid bias (BY-us). Bias means human choice or other factors that might affect the trial results. To reduce bias more, you may not know what group you’re in. This is called a single-blind trial. Only the doctor knows who is getting which treatment. Sometimes not even your doctor knows what group you’re in. This is called a double-blind trial.

Might I Get Just a Placebo?

Sometimes people worry that they will get just a placebo (pluh-SEE-bo) or “sugar pill” in a clinical trial. That does not happen in trials that compare cancer treatments. You will get either the new treatment or the best standard treatment.* In fact, you will get expert care from a team of health care professionals. This care can be better than regular treatment care.

*If no standard treatment exists, you may get a placebo. But you must agree to it first.

What Rights and Protections Do I Have?

All clinical trials have rules that protect you and your rights. It’s important that you know what they are.

Taking part in a clinical trial is up to you. It is strictly voluntary.
If you do take part, you will be carefully watched throughout the trial.
If at any time a treatment is found to be harmful, it will immediately be stopped. Then you may get other treatment from your own doctor.
You may leave a trial at any time you choose.

Informed Consent: Protecting Your Rights

Before deciding whether to take part in a clinical trial you go through a process called informed consent. Informed consent means you will get:

All the facts about a trial before you decide to participate, including:
- The exact plan for each step of the trial
- Details about the treatments and tests you may receive
- Possible side effects
- Possible benefits and risks
- How the trial may affect your daily life
Any new findings, like new risks, about your trial while it is going on

You will also get an informed consent form. It lists the key facts about the trial. If you decide to take part in a trial, you must sign this form. (However, even after you sign the form, you can still leave the trial any time you choose.)

Is Participating in a Clinical Trial Right for Me?

Taking part in a clinical trial is up to you. Family, friends, and your health care providers can help you make the decision. Knowing what the possible risks and benefits are will help you decide.

Consider the Pros and Cons

What Are the Possible Benefits?

There are a lot of good reasons to take part in a clinical trial. As a participant, you:

Will receive high-quality cancer care
May be among the first to benefit from a new treatment
Have some portions of treatment paid for by the clinical trial
Are taking an active role in the management of your care
Have the chance to improve cancer treatment and help others

What Are the Possible Risks?

There are some risks in being part of a trial, too. You may:

Receive new treatment that turns out to be not as good as the standard treatment (The risk varies depending on the phase of the trial. There is much less risk in Phase 3 trials.)
Receive the standard treatment that you would have received outside of the clinical trial (If the new treatment is proven better than the standard treatment, you will be quickly changed to the new treatment.)
Have unpleasant side effects that are different from the side effects from standard treatment
Not get all your costs covered by insurance

Ask the Right Questions

Talk to your health care providers about any clinical trial you are considering. Talk to the trial’s staff as well. Bring a list of questions and write down the answers you get. You may also want to bring someone else or a tape recorder with you.

Questions you may want to ask about being in a clinical trial include:

_____ What is the goal of the trial?

_____ What are the treatment choices in the trial?

_____ How does the new treatment work?

_____ What are the benefits?

_____ What are the risks?

_____ What other treatment choices do I have?

_____ How often and for how long will I receive the treatment?

_____ How long will the trial last?

_____ Where will the trial take place?

_____ Will all my costs be covered?*

_____ What were the results of previous trials for this treatment?

Use this space to write down other questions you may have.

Clinical Trials: Consider the Opportunity

In the past, some people thought clinical trials were just for people who had no success with other treatments. They saw clinical trials as a last resort. Now, many choose a clinical trial first. They see it as an opportunity. They want to take advantage of it. Today, people with advanced colon cancer get new, better treatments earlier. Ask about clinical trials. Ask before you start standard treatment. If you don’t, you may have to wait to be in a clinical trial.

Taking part in a clinical trial is up to you. But, you owe it to yourself to consider it. It may be one of the best choices you can make—for yourself and for others. It has been for many people.

*	All your costs may not be covered by health insurance. You can find out. Ask your health care provider or social worker.

For more information...

You can learn more about cancer research and clinical trials. Ask your doctor for more information and for trials that might be right for you. You can also get information by phone or through the Internet. Contact:

The National Cancer Institute (NCI)

Toll free:
1-800-4-CANCER
(1-800-422-6237)
TTY (for deaf and hard of hearing callers): 1-800-332-8615

www.cancer.gov

The American Cancer Society
1-800-ACS-2345
(1-800-227-2345)

www.cancer.org