Clinical Trials and Studies

A clinical trial is a scientific study conducted with volunteers. These studies are performed in order to evaluate a new treatment like drugs, devices, or biologics.

Before a new treatment is tried with human patients, it is carefully studied in the laboratory to show how to use the treatment safely and effectively. Laboratory research points out the new methods most likely to succeed but this early research cannot predict exactly how a new treatment will work with patients. With any new treatment there may be risks as well as possible benefits. There may also be some risks that are not yet known.

Participation in a clinical trial is voluntary. Your doctor or nurse may ask if you would like to enter a trial or you may hear about a trial from a fellow patient. Learn as much as you can about the trial, before you make up your mind.

There are many reasons. Often it is hope. Hope for some benefit for yourself; hope for an effective treatment; and longer and better quality of life. Just as often, patient volunteers want to contribute to a scientific research effort that may help others.

There is always a chance that a new treatment will be disappointing. However, the scientists and doctors involved in a study have reason to believe it will be as good as, or better than, current treatments. Patients in a clinical trial are among the first to receive new research treatments before they are more widely available. How a treatment will work for a patient in a trial cannot be known ahead of time. Even standard treatments, although effective in many patients, do not carry sure benefits for everyone. You should choose if you want to take part in a study or not, only after you understand both the possible risks and benefits.

Treatment Trials
Test new treatments, new drugs, innovative surgical procedures or radiation therapy, new combinations of treatments, or new methods such as gene therapy.

Prevention Trials
Test new approaches, medicines, vitamins, minerals, or other supplements that doctors believe may prevent or lower risk of illness.

Diagnostic Trials
Study designed to evaluate and improve methods for disease diagnosis or disease progression.

Observational Studies
In an observational study, investigators assess health or behavioral outcomes in groups of participants without assigning them to a specific study group. Participants continue to receive routine care.

Trial phases categorize clinical trials by study goal, stage in drug development and number of subjects.

Phase 1: Studies that are usually conducted with healthy volunteers and that emphasize safety. The goal is to find out what the drug's most frequent and serious adverse events are and, often, how the drug is metabolized and excreted.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition). Safety continues to be evaluated, and short-term adverse events are studied.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages or by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing. These studies gather additional information about a drug's safety, efficacy, or optimal use.

Researchers who conduct clinical trials are required to have a plan that describes how the trial will be conducted. Various people will be responsible for seeing that the study will be conducted in a safe manner according to the research plan. This can include the research team which may include your doctor, a nurse or coordinator, institution, and agency or company sponsoring the study. Clinical trials with experimental drugs or medical devices can also be inspected by the Food and Drug Administration (FDA). The FDA has strict guidelines and regulations that help ensure the safety of clinical trials.

Most research studies have certain requirements that must be met in order for a subject to participate. These requirements are designed to ensure the safety of the subjects and the usefulness of the research. Some studies have broad requirements such as being over 18 years old. Other studies are specific to illness, age, or lifestyle.

Participating in a clinical trial is voluntary. You can choose whether or not to participate in a study and change your mind about continuing a study at any time.

Research may involve different types of risk. A study that asks you to fill out a survey has only minor risks, such as questions that may make you uneasy. For other studies, such as taking an experimental drug, the risks can be much greater (e.g. having a bad reaction to the drug). The research team is required to explain to you any expected risks of being in the study before you decide whether or not to participate.

Not everyone who participates in a clinical trial will benefit personally. Sometimes, your participation in the clinical trial will be of benefit to society by helping researchers to learn more about a certain disease or condition. However, in studies using an experimental drug or device, there is a chance that you may benefit but this cannot be guaranteed.

Typically, a medical doctor leads a clinical trial aided by nurses and other study personnel. The person in charge of a study is called a Principal Investigator (PI). The PI is responsible for the overall conduct of the clinical trial. The PI is also responsible for assuring the safety of the participants.

Like your medical record, the information in your research record will be confidential. Information will be given only to the researchers who carry out the study or to those who make sure the study is safe and carried out the way it was planned.

Research studies can involve a wide variety of procedures, ranging from filling out surveys and questionnaires to taking experimental medicines or using experimental devices. Some research studies last only a few minutes, while others last for several years. The research team will describe to you all of the procedures that you will be asked to undergo before you agree to be in the study.

Informed consent is the process of learning the key facts about a clinical trial before you decide whether or not to volunteer. Your agreement to volunteer should be based upon a clear understanding of what will take place in the study and how it might affect you. The consent includes details about the study, tests, or procedures you may receive, the benefits and risks that could result, alternatives available should you decide not to participate, and your rights as a research volunteer.

Before you decide to volunteer for a clinical trial, you need to know as much as possible about it. If there are any issues that concern you, be sure to ask questions. You have every right to get answers to all you ask before deciding to participate in a trial. The following booklet (in English and Spanish) can help you prepare your discussion about whether a clinical trial is right for you.

English http://oprs.usc.edu/files/2013/03/Should_I_Participate_2013.pdf

Spanish http://oprs.usc.edu/files/2013/03/Public_pamph_en_Espanol_II_2013.pdf

USC Complaints, Concerns, or Reports of Violations Website http://oprs.usc.edu/about/complaints/

This website provides info on how to report a complaint, concern, or violation (anonymously if you prefer).