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What are Clinical Trials?
BACKGROUND | Phases | Participation | Questions | Find a Trial
Clinical trial is the scientific term for the study of a drug, a procedure, or a medical device. Clinical trials are performed
to determine if the product being tested is safe and effective for use in people. Doctors and other healthcare professionals
run clinical trials according to strict rules set by the Food and Drug Administration (FDA) to ensure that study participants
are treated as safely as possible. 1 There are many different types of clinical trials, which are used to find better ways to prevent, screen for, diagnose, and
treat diseases, and even to improve patients’ quality of life.
Clinical trials are designed to answer many different questions about cancer treatments 2:
- Does this treatment work?
- If so, how does it work?
- Does it work better than other treatments available?
- Are there side effects, and what are they?
- Which patients will benefit most from this treatment?
Clinical trials are often sponsored by governmental agencies. 2 For example, the National Cancer Institute sponsors about two thirds of clinical trials and is studying more than 250 anticancer
agents. Other agencies that sponsor or conduct clinical trials related to cancer are the National Institutes of Health, Department
of Defense, and Department of Veterans Affairs. Voluntary organizations, such as the American Cancer Society, also sponsor
and support clinical trials. Drug companies often join with universities and cancer centers to conduct these trials. 3
CLINICAL TRIAL PHASES
Before any new cancer treatment can be tested in humans, it is studied extensively in the laboratory. Generally, these studies
are done in human cells in the test tube (in vitro) and animals (in vivo) to determine which drugs are most likely to affect
the cancer. If the experimental results are promising, then the FDA may approve testing in humans. 3
Most clinical trials are carried out in sequential steps called phases ( Table 9-1 ). Each phase is designed to answer specific questions and builds on information from the previous phase. In Phase 1 trials, researchers learn about the side effects of a new drug by gradually increasing the dosage and analyzing patients’
responses. Based on the results, a larger study may be conducted to evaluate the potential effectiveness of the drug. 3
Phase 2 trials determine if the treatment works. 2 Researchers try to learn if the new drug has the potential to be better than current treatments. Tumor size is measured and
recorded to determine if the new drug shrinks the tumor, and blood samples are analyzed to look for side effects of the drug
and the effect of the drug on cancer markers in the blood. If enough people have a response, Phase 3 testing will proceed.
The goal of Phase 3 trials is to determine if the treatment is better than, as good as, or not as good as the accepted standard treatment. 3 In general, participants in Phase 3 trials receive the study drug as a first treatment, that is, they have had no other treatment
for their cancer. Phase 3 studies provide a more thorough understanding of the effectiveness, benefits, and side effects of
the drug. Phase 3 studies also typically determine if the new therapy is better than the current standard of care.
| Study Phase |
No. of Patients |
Patients’ Cancer Characteristics |
Primary Purposes |
| Phase 1 |
Usually less than 50 |
Usually with advanced cancer no longer responding to conventional treatments |
Safety and dosage |
| Phase 2 |
Groups of 20 to 40 |
Usually have had standard therapy but the cancer has recurred |
Safety and effectiveness |
| Phase 3 |
Hundreds |
Usually have had no prior treatment for the cancer |
Determine if study drug is as good as, better than, or not as good as standard treatment |
CLINICAL TRIAL PARTICIPATION
Because there are few effective treatments for patients with tumors that cannot be removed surgically and for patients who
have metastatic disease, clinical trials of pancreatic cancer treatments are important. They may investigate new combinations
of currently available drugs or study new drugs that have worked in other cancers. Many clinical trials are underway for patients
in all stages of pancreatic cancer. Whether to take part in a clinical trial can be a difficult decision to make. Patients
must weigh the risks and benefits of the treatment. 2 Moreover, patients need to get enough information about the trial to make a good decision. Box 9-2 lists questions to ask to help you make that decision. Remember that participation in a clinical trial is always voluntary.
If you enroll in a clinical trial, you need to know that you have certain rights. You have the right to be told 3:
- The purpose of the clinical trial
- All risks, side effects, and discomforts that might reasonably be expected
- Any benefits that can reasonably be expected
- What will happen during the study and whether any procedure, drug,or device is different from that used in standard medical
treatment
- Your available options and how they may be better than or worse than being in the clinical trial
- Medical treatments available if complications occur during your participation
As a participant you also can do the following 3:
- Ask any questions about the clinical trial before giving your consent to participate, and at any time during the clinical
trial
- Have ample time, without being pressured, to decide whether to agree to participate
- Refuse to participate before entering the trial, and leave the trial at any time after it has begun
| Box 9-1 |
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QUESTIONS TO ASK ABOUT CLINICAL TRIALS 1-4
- What is the study goal, that is, the purpose of the research?
- What are my other options?
- How much experience do the doctor and the institution have with thistreatment?
- Has the treatment been used at other cancer centers?
- If so, are the results about its safety and efficacy available?
- Is the drug already being used in another country?
- What are the known potential risks and potential benefits of the treatment?
- What exactly does the treatment consist of, and how is it carried out?
- What are the major side effects seen so far? Minor side effects?
- Who looks out for me as a study participant?
- What happens if I do not respond to this treatment?
- What happens if I respond then stop responding?
- What part of the costs am I responsible for?
- Will I need to come to this institution to receive treatment in this clinical trial? If so, how long can I expect to be here
for each treatment?
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As a participant in a clinical trial, not only will you receive excellent care,but you will be protected in various ways.
The FDA requires that you be given complete information about the study before you agree to take part,which is known as informed
consent. You will be asked to read and sign an Informed Consent Form, and will be given a copy of the signed form.This form
must be written in an understandable way for the patient. Signing the form shows that you have been given this information
and that you understand it. However, when you sign the Informed Consent Form you are not signing a contract. You may leave
the study at any time and for any reason. In addition, informed consent is an ongoing process. You have the right to be given
all pertinent information and have your questions answered at any time during your participation in the clinical trial. 1
HIGH-QUALITY CARE
IN CLINICAL TRIALS 1 |
As a participant, you will receive high-quality, individualized care. The research team will closely monitor your treatment
response, your health, and any side effects you may have. Researchers also will follow strict scientific guidelines and ethical
principles to protect participants.
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You are protected in a clinical trial in three additional ways: by Institutional Review Boards (IRBs), Data Monitoring Committees,
and FDA inspections. People from the local community, including doctors and clergy, serve on IRBs to review and monitor their
facility’s medical research that involves people. They ensure that there is the least possible risk to participants, and that
the risks are reasonable in relation to the expected benefits. 1
Data Monitoring Committees are mainly used when one treatment is being compared with another. These committees are particularly
important in testing treatments for serious or life-threatening diseases such as cancer. Experts review information from studies
to make sure they are being done in the safest way. The Committee has the power to stop a study if the treatment is harmful,
or to stop a study in order to provide the treatment to all participants when one treatment works better than another. 1
The FDA inspects records, clinics, and research sites involved in clinical trials. The FDA makes sure volunteers are being
protected and that the studies are being done properly.
| WILL I RECEIVE A PLACEBO? |
Sometimes patients think that they may receive a placebo (a sugar pill) in a clinical trial. Most clinical trials for cancer
do not use placebos. Patients usually get either the drug under study or a drug that is considered standard treatment. However,
if you have any doubts, ask.
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HOW TO FIND A CLINICAL TRIAL
Interested patients should ask members of their healthcare team about clinical trials. Many lists of ongoing clinical trials
are available on the Internet. Unfortunately, there is no one place to search for all available clinical trials. Several groups
have matching and referral services to help patients find appropriate clinical trials. (See Services & Resources for a list of organizations you can call or web sites you can search for clinical trials.)
Clinical Trials at Jefferson:
The NCI-designated Kimmel Cancer Center at Jefferson offers innovative clinical trials and a full range of supportive care
services to improve our patients' health and quality of life.
View open clinical trials for patients with pancreatic and related cancers
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REFERENCES ! Click reference number to return to text.
1. Food and Drug Administration. Department of Health and Human Services. Clinical trials of medical treatments: Why volunteer?
Washington, DC; Publication FDA 01-1294. www.fda.gov/opacom/lowlit/cltr.html. Accessed June 20, 2007.
2. American Cancer Society. Clinical trials. www.cancer.org/docroot/ETO/content/ ETO_6_3_Clinical_Trials_-_Patient_ Participation.asp.
Accessed April 16, 2007.
3. Eyre HJ, Lange DP, Morris LB. Informed Decisions: The Complete Book of Cancer Diagnosis, Treatment, and Recovery. 2nd ed.
American Cancer Society–Health Content Products. Atlanta, GA; 2002.
4. Getz K, Borfitz D. Informed Consent. A Guide to the Risks and Benefits of Volunteering for Clinical Trials. Boston, MA: Thomson;
2003.
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