Clinical trials – research studies that involve patients – represent a crucial step in the advancement of medicine and the development of potentially life-saving drugs and treatments. Keck School of Medicine faculty members lead and/or participate in many of the nation’s largest multi-center clinical trials, as well as in many unique smaller trials. Although participating in a clinical trial is not the right choice for everyone, many patients receiving care in one of the Keck-affiliated academic hospitals have chosen to become involved in clinical trials.

At the Keck School, clinical research focuses on testing new techniques and therapies for disease, optimizing existing treatments, discovering new disease prevention methods and developing ways to improve the quality of life for both healthy individuals and those living with disease.

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 multiple sclerosis or heart disease. Some are “adjuvant,” such as those testing hormones to prevent 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 own genes to determine what genetic makeup responds best to particular treatments.

What are the Benefits of 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, the patient is closely observed, both by the physician and a research nurse. Each trial has a "protocol" that specifies how and when patients are seen, given their medication and monitored with blood and other diagnostic tests.
• Advancing Medicine
  Although the main motivation to enter a clinical trial is the possibility of obtaining the best treatment, participants are also advancing the war on cancer and other diseases.

What are the Phases of Clinical Trials?

Clinical Trials are divided into four "phases." Which trial is offered to an individual patient depends upon the stage of their disease, 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 and how much can be given safely. Phase I trials are usually offered only to patients with advanced disease who would not be helped by other known treatments. Some patients are helped by these treatments, although in this early stage physicians don’t really know if the treatment will be effective.
• 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've received the investigational agent. At best they are allowed to get a new drug they wouldn't be able to get otherwise that may turn out to be better for their disease.
• If a treatment has shown activity against a particular disease, it is moved up to Phase III, where it is either added to existing treatment or compared to the standard treatment.
• Phase IV refines the treatment to become part of standard care.

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. Many represent simply a new approach, such as dosing or schedule changes, or addition to existing standard treatments, such as a medication that may enhance the effectiveness of a standard drug or reduce a toxic side effect. 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, but it must be first 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 others in the community who additionally approve the proposal for safety and ethical issues.

Aside from reviewing the science and safety and efficacy, the IRB gives serious consideration to the proposed trial's "informed consent" — the document that physicians will give to patients explaining the trial and its potential risks.

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