University of Southern California
USC Norris Comprehensive Cancer Center

Frequently Asked Questions About Clinical Trials

What is a clinical trial?
What are the types of clinical trials?
How do clinical trials work?
What are the benefits of participating in a clinical trial?
What are the possible risks of participating in a clinical trial?
What are the phases of clinical trials?
How are clinical trials monitored?
Can a clinical trial be stopped?
What do I need to know when deciding whether to participate in a clinical trial?
How can I volunteer to participate in a clinical trial?

What is a clinical trial?
A clinical trial is a research study that involves human volunteers with specific health conditions. The trials allow the examination of a proposed drug or therapy in a controlled environment to determine its safety and effectiveness in treating patients with certain conditions.

What are the types of clinical trials?
The majority of clinical trials are therapeutic, offering new treatments to those who have advanced diseases such as late-stage cancer. Some are adjuvant, such as those testing hormones to prevent the recurrence of breast cancer. Prevention trials test agents that may help reduce the likelihood of developing diseases for those at high risk. In translational trials, researchers examine a patient's genes to determine what genetic makeup responds best to particular treatments. Observational trials address health issues in large groups of people of populations in natural settings.

How do clinical trials work?
In many clinical trials, one group of patients will be given an experimental drug or treatment, while patients known as the control group are given either a standard treatment for the illness or a placebo. A placebo is an inactive pill, liquid or powder that has no treatment value. Comparing the two groups allows researchers to evaluate the effectiveness of the study drug or therapy. In "blind" or "masked" studies, patients and sometimes the study staff do not know if they are receiving the experimental treatment or a placebo.

What are the benefits of participating in a clinical trial?
There are numerous potential benefits to participating in a clinical trial:

Aggressive treatment
For patients with advanced disease, clinical trials offer some of the most aggressive options for treatment.

Early access to treatments
When an agent being tested in a clinical trial proves superior, patients benefit by having received a treatment they wouldn't have been able to receive otherwise.

Close monitoring
Because clinical trial treatments are experimental, each trial has a protocol, or action plan, that specifies how and when patients are seen, given their medication and monitored with blood and other diagnostic tests. Patients involved with trials will interact closely with a physician or scientist and a research nurse.

Advancing medicine
Although the main motivation for participating in a clinical trial is the possible benefits realized by the patient, participants are also advancing progress in the discovery of causes and potential treatments and cures for cancer.

What are the possible risks of participating in a clinical trial?
Risks vary by each clinical trial and should be explored fully with your health care providers and the study administrators before you consent to participate. These may include unpleasant, serious or life-threatening side effects, the possibility that the study drug or treatment is not effective in treating your cancer or the inconvenience of making additional trips to the study center or participating in additional tests as a condition of enrolling in the study.

What are the phases of clinical trials?
Clinical trials are divided into a series of four steps, or phases. The phase at which time certain patients become involved in a trial depends upon the stage of their cancer, prior treatment and the specific eligibility criteria of the trial.

  • A Phase I trial is a new treatment that has not been tested, and researchers are looking for the best way to administer the treatment, determine how much can be given safely and identify side effects. Phase I trials are usually offered only to patients with advanced disease who would not be helped by other known treatments.
  • A Phase II trial takes the treatment one step further, assessing the activity of a particular therapy in a disease, often building upon leads from the Phase I trial. While patients are generally required to be previously untreated, participation in a Phase II trial doesn't usually preclude the patient from getting the standard treatment after they have received the investigational agent or study drug/therapy.
  • If a treatment has shown activity against a particular disease, it is granted the status of a Phase III trial, where it is either added to existing treatment or compared to the standard treatment. Phase III trials typically involve larger groups of patients in order to confirm the treatment's effectiveness and monitor side effects.
  • Phase IV refines the treatment to become part of standard care. This phase typically takes place after a drug or treatment has been marketed to further evaluate its effect on different populations of patients and examine long-term use.

How are clinical trials monitored?
Clinical trials are rigorously regulated. As with any new medication or surgical procedure, treatments are first studied thoroughly in the laboratory. If basic research establishes that a new method or chemical seems likely to succeed safely and effectively in patients, it can be submitted for approval for a clinical trial.

Not only must the submission meet regulatory guidelines established by organizations such as the National Cancer Institute (NCI) and the Food and Drug Administration (FDA), but also it must first be reviewed for scientific merit by the Hospital Clinical Investigations Committees. It is then submitted to the USC Health Sciences Institutional Review Board (IRB), a panel of 36 scientists, researchers and representatives in the community who additionally approve the proposal for safety and ethical issues.

Aside from reviewing the scientific basis for the trial, as well as its safety and efficacy (the ability of the treatment or drug to produce its intended effect), the IRB gives serious consideration to the proposed trial's "informed consent" - the document that physicians give to patients explaining the trial and its potential risks.

All clinical trials must have a protocol, or action plan, which describes in detail what will be done in the study, how it will be conducted and why the various portions are necessary.

Once approved, clinical trials are monitored through the hospitals' Clinical Investigations Support Offices, where research nurses follow patients closely and data managers track their progress.

Can a clinical trial be stopped?
The Institutional Review Board can stop a study if it appears to be causing unexpected harm to participants, or if there is evidence that the risks outweigh the benefits. An IRB can also stop a study if there is clear evidence that the new treatment is effective, in order to make it widely available.

Patients participating in a clinical trial may leave the study at any time, since their inclusion is voluntary.

What do I need to know when deciding whether to participate in a clinical trial?
The informed consent that you will receive from the physician should explain the major elements of the trial and answer questions you might have about the motivations for the study, what is required of you in order to participate, potential risks and benefits, alternatives for treatment and potential costs. Questions you may wish to ask include:

  • Why is the study being done?
  • Has the drug or treatment in this study been tested before?
  • Who is sponsoring the study?
  • What will happen during the study?
  • What am I required to do to participate in the study?
  • Will I need to be hospitalized to participate? If not, how often will I need to come to the study center?
  • What are the possible risks or discomforts?
  • What are the possible benefits of taking part in the study?
  • What other treatment alternatives do I have?
  • Will I receive the study drug or treatment during the clinical trial, or is it possible that I may be part of a control group receiving a placebo instead?
  • Are there any costs involved in participating in the study or receiving the care as part of the study?
  • Will I receive any compensation for participating in the study?
  • What happens if I am injured as a result of my participation?
  • What do I do if I need emergency care during the study?
  • What are my rights as a participant in the study?
  • What will be done with the information gathered from the study?
  • Will I be able to get information about the results of the study?
  • Who can I call with questions or problems?

Once you have had your questions answered to your satisfaction, you can consult with your health care providers, family and friends to decide whether participating in a clinical trial might beneficial for you.

How can I volunteer to participate in a clinical trial?
To find out more information about clinical trials at USC/Norris, visit the clinical trials database or contact the Clinical Investigation Support Office at (323) 865-0451.

Please keep in mind that all clinical trials have specific guidelines about who can participate. Guidelines are based on such factors as age, type of disease, medical history and current medical condition. So while volunteering for a clinical trial is appreciated, it is important to recognize that it is not always possible to include all volunteers in a trial.