What Are Cancer Clinical Trials?

In the discussion of cancer treatment, the term cancer clinical trials appears frequently.  Also called research studies, cancer clinical trials test many types of treatment such as new drugs, new approaches to surgery or radiation therapy, new combinations of treatments, or new methods. Cancer clinical trials are the first step in testing a new treatment in humans and their goal is to find better ways to treat cancer.

Cancer clinical trials include research at four different phases.  Each phase answers different questions about the new treatment:

Cancer Clinical Trials: Phase I

The questions being explored in Phase I cancer clinical trials are:

• What is the best way to give a new treatment?
• Can this medication be given safely to humans?
• What is a safe dose?

These cancer clinical trials involve a limited number of patients who may not be helped by other known treatments.

Cancer Clinical Trials: Phase II

In Phase II of cancer clinical trials, the focus is on learning whether the new treatment has an anticancer effect on a specific cancer.  Additional information regarding the side effects of the treatment is also obtained.  A small number of people are included because of the risks and unknowns involved. 

Cancer Clinical Trials: Phase III

In Phase III of cancer clinical trials, a comparison of the results of people taking a new treatment is done with the results of people taking a standard treatment. Some of the questions being asked in a Phase III trial are:

• Which group has better survival rates?
• Which group has fewer side effects?

Persons are assigned at random (a process similar to flipping a coin) to either the new treatment (treatment group) or the current standard treatment (control group).  Randomization helps to avoid bias (having the study's results affected by human choices or other factors not related to the treatments being tested).  Some cancer clinical trials compare a new treatment with a placebo (a look-alike pill/infusion that contains no active drug).  However, a person is told if this is a possibility before deciding whether or not to take part in a study.

Comparing similar groups of people taking a different treatment for the same type of cancer is another way to make sure that the study results are real and caused by the treatment rather than by chance or other factors. These cancer clinical trials may include hundreds to thousands of people from different centers around the country.

Cancer Clinical Trials: Phase IV

In Phase IV of cancer clinical trials, also called post-marketing studies, cancer clinical trials are conducted after a treatment has been approved.  The purpose of these cancer clinical trials is to provide an opportunity to learn more details about the treatment, such as:

• The mechanism of action
• Fine points regarding toxicities
• Fine points regarding quality of life
• Questions that may have come up during other phases of cancer clinical trials.

These "post-marketing" trials may be conducted in a Phase I, II or III format.

What Happens in Cancer Clinical Trials?

In cancer clinical trials, patients receive treatment and doctors carry out research on how the treatment affects patients.  A person's progress is closely monitored during the clinical trial.  Once the treatment portion of the clinical trial has been completed, patients may continue to be followed in order to gather information regarding specific endpoints. These endpoints are defined prior to the study being started and may include time to disease progression and/or overall survival. 

While cancer clinical trials have risks for the people who take part, each study also takes steps to protect patients.  Cancer clinical trials are often the best or optimal treatment approach available.

Informed Consent in Cancer Clinical Trials

Informed Consent is an ongoing process during cancer clinical trials in which all of the available information about the specific trial is discussed with the person participating in the trial.  The doctor or nurse reviews the treatment plan, including potential risks and benefits of the treatment with the participant. This information is also written in a document (consent form) which is presented to the participant before treatment can begin.

After the potential study participant reads the document, an opportunity to ask questions about any parts of the form that are unclear is given.  If the person agrees to participate in the study the consent form is signed.  Signing the form indicates that the study participant read the form and the doctor or study nurse answered any questions about the information contained in the form, which may have been unclear. 

Signing a consent form does not mean a person must stay in the study.  In fact, people may leave cancer clinical trials at any time.  If a person chooses to leave the study, a chance to discuss other treatment options and care with their doctor is given.  A person may choose not to participate in the study, if so, their care will not be affected in any way.

Protocols in Cancer Clinical Trials

The protocol is the action plan for cancer clinical trials.  The plan states what will be done in the study and why. It outlines how many people will take part in the study, what types of patients may take part, what tests they will receive and how often, as well as the treatment plan.  Each doctor that treats patients in the study uses the same protocol, and must follow the guidelines that are specified.  The federal Food and Drug Administration (FDA) has general guidelines that must be followed by any physician or institution conducting cancer clinical trials.  Before the FDA can approve a treatment, the study results are audited to ensure the trial was conducted safely and according to these guidelines.

For patient safety, each protocol must also be approved by the organization that sponsors the study.  The Scientific Review Committee, a group of individuals from the institution, including physicians, scientists, nurses, administrators, review the protocol for scientific merit and feasibility of the protocol.  The Institutional Review Board (IRB), of the hospital, must also approve it.  This board includes consumers, clergy, and health professionals.  They review the protocol to try to be sure that the research will not expose patients to extreme or unethical risks.

Eligibility criteria are guidelines from the protocol, which describe the characteristics that all participants in the study must have.  These criteria differ from study to study, depending on the purpose of the research.  Examples are; age, gender, the type and stage of cancer; and whether cancer patients who have had prior cancer treatment or have other health problems can participate.

Possible Benefits of Cancer Clinical Trials:

• Cancer clinical trials offer high quality cancer care. 
• You may be among the first to benefit from a new treatment.
• By looking at the pros and cons of cancer clinical trials and other treatment choices, you are taking an active role in a decision that affects your life.
• You have the chance to help others and improve cancer treatment.

Possible Drawbacks of Cancer Clinical Trials:

• New treatments being studied are not always better than or even as good as, standard care.  They may have side effects that are unexpected or that are worse than those of standard care.
• Even if a new treatment has benefits, it may not work in your case.  Even standard treatments, proven effective for many people, do not help everyone.
• If you receive the standard treatment rather than the new treatment being tested, it may not be as effective as the new approach.
• Insurance companies do not always cover all patient care costs in a study.  What is covered varies by plan and by study. 

Questions You Should Ask Your Healthcare Provider:

• What is the purpose of the study?
• What has previous research of this treatment shown?
• What is likely to happen in my case with, or without the treatment?
• Are there standard treatments for my type of cancer?
• How does this study compare with standard treatment options?
• What Phase is this cancer clinical trial?
• What are the possible short and long-term risks, side effects and benefits of the treatment?
• What kinds of treatments, medical tests, or procedures will I have during the study? And how do they compare with what I would receive outside of the study?
• How long will the study last?  Will there be a follow-up after the study?
• Where will my treatment take place? Will I have to be in the hospital?
• How will I know the treatment is working?
• How could the study affect my daily life?
• Will my records be kept confidential?
• Will my insurance pay for the treatments?
• If I decide to withdraw from the study, will my care be affected?  Will I need to change doctors?

More Chemotherapy Information:

Chemotherapy Terms Chemotherapy Protocols - How Chemotherapy Works How Chemotherapy Is Given How Doctors Decide Which Chemotherapy Drugs To Give How Long Chemotherapy Is Given How To Tell If Chemotherapy Is Working Cancer Cells & Chemotherapy Types of Chemotherapy Targeted Therapy The Immune System About Immunotherapy Hormone Therapy Chemoporotective Agents Chemotherapy Resistance Short & Long Term Side Effects of Chemotherapy Nadir Cancer Clinical Trials

Chemocare.com is designed to provide the latest information about chemotherapy to patients and their families, caregivers and friends. For information about the 4th Angel Mentoring Program visit www.4thangel.org