Clinical trials are research studies involving people. They are the final step in a long process that begins with preliminary laboratory research and animal testing. Clinical trials try to answer specific scientific questions to find better ways to prevent, detect, or treat diseases or to improve care for people with diseases.
Many of today's most effective cancer treatments are based on previous study results. Because of progress made through clinical trials, many people treated for cancer are now living longer. The more people who participate in clinical trials, the faster critical research questions can be answered that will lead to better treatment and prevention options for all cancers. We will never know the true effectiveness of a cancer treatment or a way to prevent cancer unless more people are involved in clinical trials.
In the past, clinical trials were sometimes seen as the last resort for patients who had no other treatment choices. This is not true; there are many clinical trials for individuals whose cancer has not spread.
Few people with cancer take part in cancer clinical trials : Enormous improvements in treating childhood cancer have come about as the direct result of clinical trials; more than 60 percent of U.S. children with cancer participate in clinical trials. In 2000, more than 70 percent of children with cancer were alive 5 years after diagnosis, compared to only 55 percent in the mid-1970s. In contrast, fewer than 5 percent of U.S. adults with cancer participate in clinical trials-far fewer than the number needed to answer the most pressing cancer questions quickly.
According to a 2000 Harris Interactive survey, most people with cancer were either unaware or unsure that participation in clinical trials was an option for their treatment, and most of them said they would have been willing to enroll had they known it was possible.
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There are several types of clinical trials. Each one is designed to answer the following questions. Treatment trials are the most commonly used in cancer research.
Treatment trials • What new treatment approaches can help people who have cancer? • What is the most effective treatment for people who have cancer? Prevention trials • What approaches can prevent a specific type of cancer from developing in people who have not previously had cancer? Early-detection/screening trials • What are new ways of finding cancer in people before they have any symptoms? Diagnostic trials • How can new tests or procedures identify cancer more accurately and at an earlier stage? Quality-of-life/supportive care trials • What kind of new approaches can improve the comfort and quality of life of people who have cancer? return to top
Treatment trials • What new treatment approaches can help people who have cancer? • What is the most effective treatment for people who have cancer?
Prevention trials • What approaches can prevent a specific type of cancer from developing in people who have not previously had cancer?
Early-detection/screening trials • What are new ways of finding cancer in people before they have any symptoms?
Diagnostic trials • How can new tests or procedures identify cancer more accurately and at an earlier stage?
Quality-of-life/supportive care trials • What kind of new approaches can improve the comfort and quality of life of people who have cancer?
Once laboratory studies show that a new approach has promise, a phase 1 trial can begin. A phase 1 trial is the first step in testing a new cancer agent in humans. In these studies, researchers look for the best way to give people the new agent (for example, by pill or by injection), how often it should be given, and what the safest dose is. These studies also include special laboratory tests such as blood tests and biopsies to evaluate how the new agent is working in the body. Phase I cancer trials involve small groups of people with cancer.
Phase 2 trials continue to test the safety of the new agent, and begin to evaluate how well it works against a specific type of cancer. In these trials, the new agent is given to groups of people with one type of cancer or related cancers, using the dosage found to be safe in phase 1 trials. Phase 2 cancer trials usually have less than 100 participants.
Phase 3 trials focus on learning how a new treatment compares to standard, or the most widely accepted, treatment. Researchers want to learn whether the new treatment is better than, the same as, or worse than the standard treatment. In phase 3 trials, participants have an equal chance to be assigned to one of two or more groups (also called "arms"). In a study with two groups:
The process of assigning participants to groups is called randomization.
Phase 4 trials are used to further evaluate the long-term safety and effectiveness of a treatment. Less common than phase 1, 2, and 3 trials, phase 4 trials take place after the new treatment has been approved for standard use.
Summary of Clinical Trials Phases
Phase
Number of Participants
Goals
I
II
III
IV
An endpoint is what researchers will measure to evaluate the results of a new treatment being tested in a clinical trial. Research teams establish the endpoints of a trial before it begins.
It is important to note that endpoints differ, depending on the type and phase of the clinical trial. Examples of endpoints are:
The NCIC CTG, pharmaceutical companies, medical institutions, and other organizations sponsor clinical trials. Regardless of sponsor, clinical trials take place at universities, large medical centers, small hospitals, and doctors' offices. Individual physicians at cancer centers and other medical institutions can also sponsor clinical trials themselves.
NCIC CTG-Sponsored Clinical Trials The NCIC CTG (National Cancer Institute of Canada Clinical Trials Group) is a cooperative oncology group which carries out clinical trials in cancer therapy, supportive care and prevention across Canada and internationally. It is one of the national programmes and networks of the National Cancer Institute of Canada (NCIC), and is supported by the NCIC with funds raised by the Canadian Cancer Society (CCS).
If you are a cancer patient, your doctor can explain how this information applies to you, or you can call the Cancer Information Service of the Canadian Cancer Society at 1-888-939-3333. CancerNet also contains PDQ (Physician Data Query) information for patients.
Clinical Trials Cooperative Group Program Clinical trials are often conducted through cooperative clinical trial groups, which are networks of institutions that jointly carry out large clinical trials following the same protocols. Members of these groups include university hospitals, cancer centers, community physicians, and community hospitals. Cooperative groups develop and conduct new clinical trials that follow national priorities for cancer research. They conduct phase 3 trials as well as phase 2 trials. Pharmaceutical and Biotech Programs Pharmaceutical and biotech companies conduct their own trials, both locally and nationally. They may have partners such as universities, hospitals, NCIC GTC, or local doctors. These trials are subject to the companies' own review panels and to an Institutional Review Board (IRB), which may be local or national in scope.
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