What is a research study?
A research study is designed to test the safety and/or effectiveness of drugs, devices, treatments or preventive measures in humans.
What is the difference between a Phase I, Phase II, Phase III and Phase IV clinical trial?
A clinical trial is a research study designed to test the safety and/or effectiveness of drugs, devices, treatments or preventive measures in humans. Research studies can be divided into four categories or "phases."
Phase I trials are the first experiments using an investigational new drug in humans. Normally, healthy participants are enrolled in Phase I studies; however, participants with the disease being studied may, under certain circumstances, be enrolled into the trial. Phase I studies are designed to determine how the drug is broken down in the human body and how it interacts with the human body. Phase I studies reveal some of the side effects associated with increasing doses. When possible, some Phase I studies provide early evidence of effectiveness. Researchers use information from Phase I studies to design Phase II studies. The total number of participants included in Phase I studies varies with the drug, but is generally a small number of participants or a few dozen.
Phase II trials continue to test the safety of the drug, and begin to evaluate how well the new drug works. Researchers collect additional safety and effectiveness information, study short-term side effects and risks, and collect additional information about the proper dose and dosing schedule. Phase II studies are closely monitored and typically involve control groups.
Phase III trials are done after Phase I and Phase II studies have shown some evidence of effectiveness. They are longer-term research studies and are intended to gather the additional information about effectiveness and safety that is needed to evaluate the overall benefit/risk relationship of the drug. At the end of most Phase III trials the new drug will be found to be inferior, equal or superior to the standard treatment.
Phase IV trials are done after the new drug has been approved by the U.S. Food and Drug Administration. Researchers continue to gather information about an approved drug's risks, benefits and best uses associated with large-scale usage in "real-life conditions." Phase IV studies can also include trials of different doses or schedules of administration, other stages of disease, cost studies, quality-of-studies or use of the drug over a longer period of time.
What is a human subject?
A human subject is a volunteer participant who is or becomes involved in a research study. A subject can be either someone who is healthy or someone with a particular medical condition.
What is an investigator?
An investigator is a person who initiates and carries out a research study. He or she is responsible for study procedures and treatments. Studies often have a "Principal Investigator" who is responsible for overseeing the conduct of the study.
What is a sponsor?
A sponsor is a person or funding source who initiates a research study, but does not actually conduct the study. Sometimes a sponsor will be a pharmaceutical company, but it may also be a federal agency or Rush University Medical Center.
What is an informed consent document?
Informed consent is required for participation in all research studies involving human subjects. In the informed consent, potential subjects are given information about what is involved in the study, including possible benefits and risks. Potential subjects are encouraged to ask as many questions as they like. Below are some examples of the type of questions to consider before signing a consent form.
What is the purpose of the study?
What type of tests and treatments are involved?
Are there any costs involved?
What are potential side effects?
How could this affect my daily life?
What is involved and for how long?
What other choices do I have? What are their advantages and disadvantages?
What are the benefits of volunteering to take part in clinical research?
By participating in clinical research you may provide important scientific information for developing new disease treatments and further medical knowledge.
Are there risks or side effects in clinical studies?
There may be risks with any clinical study. The risks and benefits are reviewed in detail in the informed consent process. You are encouraged to ask questions throughout the consenting process and while you are in the study. There are no guarantees that the drug or device being studied will be effective. The principal investigator or study coordinator will explain the potential risks and the potential benefits before you sign the consent form and join the study.
Can I quit or leave a study at any time?
Yes. Participation in a research study is voluntary. You may leave at any time for any reason. You will need to speak to the study investigator regarding any safety precautions that might be required before or upon your departure from the study. The investigator can also withdraw you from the study if new potential risks are identified and/or if you are no longer benefiting from the treatment.
What is an investigational drug or device?
An investigational new drug means a new drug or biological product that is not approved by the Food and Drug Administration (FDA) for a particular condition and is being used in a study on that condition. An investigational device is a device that is not approved by the FDA and is being used in a study.
What is an experimental group?
An experimental group is the group that receives the experimental drug, device, treatment or intervention.
What is a control group?
Participants in the control group are used as a standard of comparison. For example, a study may have two experimental groups and a control group. The experimental group is given the experimental treatment under study, while the control group may be given the standard treatment for the illness or a placebo for the study. At the end of the study, the results of the two groups are compared.
What is a placebo?
A placebo is an inactive substance that is used for testing against active treatment. It will often look like the active treatment, but will not contain active ingredients. The results of the active treatment are compared to the results from the placebo to better understand the actions of the active treatment.
What is the difference between a treatment study and a prevention study?
A treatment study tests new treatments (such as new drugs or devices, new combinations of treatments and/or approaches to surgery or radiation therapy). A prevention study tests new approaches that doctors believe may lower the risk of a condition (such as vitamins, minerals, supplements or other interventions).
What is a blinded study? What is the difference between a single and double-blinded study?
A blinded study is a study where you, the participant, will not know which treatment or procedure you are receiving. In a single-blind study, the researcher will know which treatment or procedure the participants are receiving, while in a double-blind study, neither the participant nor researcher will know which treatment or procedure the participants are receiving. It is done so that expectations about the treatment will not influence the study results.
What does randomization mean in research?
A randomized study is one where the study groups (experimental and/or control) are assigned by chance, like the flip of a coin or the roll of dice.
How does randomization prevent bias in research?
Research subjects are randomized in clinical trials so that bias does not weaken the study results. Bias refers to human choices, beliefs or any other factor besides those being studied that can affect a study's results. If physicians or participants themselves choose the group, assignments might be personally influenced and therefore unevenly slanted toward one side or the other.
Who can I contact with questions or to obtain more information about the research study?
All research subjects receive a copy of the informed consent document when they enroll. This document includes contact information for the Investigator and/or the investigator's staff.
Who can I contact with questions about my rights as a research subject?
Questions about your rights as a research subject should be directed to the Rush Division of Research Regulatroy Operations at (800) 876-0772. You may also contact this group to discuss problems, concerns with the study and/or to offer input.