What is a clinical research trial?
A clinical trial is a research program conducted with participants to evaluate a new medical treatment, drug or device. The purpose of clinical trials is to find new and improved methods of treating different diseases and special conditions.
There are four phases of a clinical trial:
- Phase I: usually the first time a new treatment, drug or device is used in humans. In this case, the treatment is given to a small number of participants at a very low dose or setting, and increased over time according to the response. The purpose is to determine the best way to give the new treatment and how much of it can be given safely.
- Phase II: determine the effect of a research treatment on a particular disease or condition. Phase II trials involve the development of a protocol, or plan about how the trial will be conducted and how the data will be analyzed. These plans are submitted to a an Institutional Review Board (IRB), who must approve the protocol before the clinical trial begins. The IRB is a group of health professionals and non-medical persons whose job it is to review and monitor clinical research to protect the safety and rights of participants. Some research is also reviewed and monitored by federal agencies (such as the National Institute of Health - NIH, or U.S. Food and Drug Administration - FDA). There may or may not be a control group in a Phase II clinical trial. Control groups receive standard, current care. Treatment groups receive the experimental therapy.
- Phase III: compare the new treatment with the standard treatment on a larger group of participants to determine effectiveness, dosing, and optimal characteristics of a treatment, device or drug. The same review and approval process takes place in a Phase II clinical trial. There is usually a control group and a treatment group. The participant group is chosen based on randomization, or assigning a participant into a group based on chance.
- Phase IV: This phase of research is conducted after approval has been given by the FDA. The purpose of Phase IV research is to learn more about how the treatment, drug or device works in a larger population of participants or to determine clinical advantages of a specific treatment.
What does it mean to participate in clinical research?
Who can participate?
Every clinical trial is designed to look at a specific set of participants. Each study enrolls participants with certain conditions and symptoms. If you fit the guidelines, you may be able to participate. In some instances, you may be asked to undergo certain tests to make sure you are appropriate for the study.
What are the advantages?
During a clinical trial, Investigators use the best treatment available as a standard to evaluate new treatments. The new treatments are at least as effective or possibly more effective than standard. If you participate:
- You may receive a new treatment before it is widely available to the public
- You can provide researchers with the information they need to continue developing new procedures and treatment methods
- Your treatment costs may be decreased (many of the tests and Investigator visits that are directly related to the study are paid for the company or agency conducting the clinical trial).
Could there be side effects?
Yes. Side effects will depend on the type of treatment and the participant's condition. All of the risks and side effects of the treatment being researched are not known at the beginning of the clinical trial. Since this is the case, there may be unknown side effects, as well as hoped for benefits.
It is important to note that most treatments, as well as the condition itself, have potential side effects. In clinical trials, most side effects from the treatment are temporary and go away once the treatment is stopped. participants will be informed of any known possible side effects that occur while they are participating in a trial.
What is informed consent?
- Informed consent means that as a participant, you are given all the available information so that you understand what is involved in a specific clinical trial. Your doctor or nurse should explain the treatment to you, including benefits and risks.
- You will be given an informed consent form. Read it and consider it carefully. Ask the Investigator or nurse to explain any parts of the form that are not clear.
- You are free to decide whether or not to take part in the trial. If you decide to take part in the study, you will sign the consent form. If you do not want to participate in the trial, you can say "No." If you choose not to participate in the trial, your care will not be affected in any way.
- After you sign the form, you will receive a copy. It contains important information that you may refer to during the trial.
Questions to ask your doctor
If you are thinking about taking part in a clinical trial, find out as much as possible about the study before you decide to participate. Here are some questions you may want to ask your doctor or nurse?
- What is the purpose of the study?
- What have previous research of this drug or treatment shown?
- What kinds of tests and treatments does the trial involve? How are these tests given?
- What is likely to happen in my case with, or without, this new research treatment? (How does this research study compare with standard treatment options?)
- What phase is this clinical trial?
- How could the clinical trial affect my daily life?
- What side effects can I expect from the clinical trial? (Remember, there can also be side effects from standard treatments and from the disease itself)
- How long will the study last?
- Will the clinical trial require extra time on my part?
- Will my records remain confidential?
- Will the study cost me anything?
- Will I have to be hospitalized? If so, how long and how often?
- If I agree to withdraw from the clinical trial, will my care be affected? Will I need to change doctors?
(Adapted from Clinical Trials: A guide for patients, The Cleveland Clinic Foundation, 1995)
For more information:
Information on this page from, "Should I Enter a Clinical Trial? A Patient Reference Guide for Adults with a Serious or Life-Threatening Illness." Written by ECRI *(formerly the Emergency Care Research Institute) with an educational grant from Pfizer, Inc. to the American Association of Health Plans, 2002.