Clinical Trials

What are Clinical Trials?

Clinical trials are studies that evaluate the effectiveness of new drugs or treatment strategies. The development of more effective cancer treatments requires that new and innovative therapies be evaluated with cancer patients. In oncology, clinical trials are especially important because, in the absence of high cure rates, nearly all therapeutic approaches are developmental in nature. All new cancer drugs that are currently available in the United States were once only available in clinical trials. Patients should decide whether or not participation in a clinical trial is an appropriate treatment option as early in their evaluation as possible because, once treatment of their cancer has been initiated, patients may become ineligible to participate in some clinical trials.

Clinical trials are designed to answer questions about treatment of various types of cancer. Trials can either be in-house, where the trial takes place in a single institution, or multi-institutional, where multiple cancer treatment facilities participate in the study. The most common types of clinical trials are multi-institutional trials because it often takes too much time to accrue a sufficient number of patients in a trial to design it as a single-institution study. However, the advantage of a single-institution study is that there tends to be less variability in treatment technique, which can affect the outcome.

Clinical trials for cancer patients may be phase I, II, or III. In a phase I or phase II trial, the patient participates in an investigational type of treatment but is not randomly assigned to participate in one of two or more different treatments. Phase I trials usually are done to determine the safe dose of a new treatment drug or regimen. A phase II trial is designed to determine in a small group of patients whether a new treatment is effective in treating a certain disease. In a phase III trial, the patient is randomized (randomly assigned, by chance, similar to the toss of a coin; usually done by computer) to one of two or more different treatments, or “treatment arms,” that the study is designed to compare. For example, the comparison may be between a standard treatment and a new treatment, or between a single treatment modality and a combination therapy. Usually a phase III trial includes a larger number of patients. For the patient and physician to participate in a phase III trial, it is necessary that the physician does not have any strong supposition as to which of the treatment arms might be more advantageous. Controlled clinical trials are critical to answering many of the important treatment questions that exist today and will arise in the future.

• Current clinical trials at the University of Florida Health Cancer Center
• Current clinical trials at the University of Florida Proton Therapy Institute

Clinical Research Groups

In-House Protocols

In addition to participation in multi-institutional clinical trial groups, the Radiation Oncology Department participates in in-house protocols (sets of instructions for treatment, or treatment plans) that are used at the University of Florida. Some involve only the Radiation Oncology Department; many others represent joint effort by two or more departments. Most of these protocols are considered “standard of care” (and are not detailed here), but some are investigational.

Observational Studies

Studies where investigators assess health outcomes in participant groups according to a research plan or protocol are called observational studies. Participants may receive new drugs or procedures as part of their routine medical care, but participants are not assigned to specific new drugs or treatment strategies by the investigator, like in a clinical trial.

Outcome Studies

Outcome studies are necessary to determine the effectiveness of treatment and the severity and number of complications associated with cancer treatment. To perform an outcome study, it is necessary to see patients periodically in clinic after they complete treatment, when possible, and to obtain follow-up information on patients who are no longer able to come in for check-ups. Patients with various types of cancer have their records updated periodically at the University of Florida. Data from these records are analyzed to find out what treatments have been effective as well as those that are less than optimal so that our treatment techniques can be improved.

Outcome studies have been performed annually by all of the faculty and residents in Department of Radiation Oncology at the University of Florida since approximately 1970. During the Department’s annual research seminar in the spring, the results of these studies are presented to visiting radiation oncologists and others from all over Florida and other parts of the country.

Many times these studies are also presented at national or international meetings and published in peer-reviewed journals.