What are Clinical Trials?

Research on humans is known by many names: research study, clinical trial, experimental treatment, etc.

Clinical Trials Are Research, Not Proven Treatment

Participating in clinical trials is primarily meant to benefit future patients, not necessarily participants in the trial. Typically researchers hope that participants will benefit from a trial, and there may be information from other clinical trials to suggest that a benefit is possible or likely. However, it is not known if you will benefit by participating in a clinical trial – that is why they are doing the research (for example, the Women’s Health Initiative study of Estrogen Plus Progestin in Postmenopausal Women noted an increased risk of stroke).

Participating in a Clinical Trial Is Voluntary

You may choose not to participate in a trial or to discontinue your participation in a trial at any time without it affecting your relationship with the researcher, other medical staff, Shands hospital, and/or the University of Florida.

For more information about the rights of people who participate in clinical trials, please contact the University of Florida Health Science Center Institutional Review Board at (352) 273-9600.

Information about Clinical Trials

What is a clinical trial?
According to the government’s web site, ClinicalTrials.gov, a clinical trial is defined as “A research study to answer specific questions about vaccines, new therapies or new ways of using known treatments. Clinical trials (also called medical research and research studies) are used to determine whether new drugs or treatments are both safe and effective. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people.”1Back to top
Are clinical trials proven treatment?
No, clinical trials are not proven treatment. Clinical trials are research and the purpose of all clinical trials is to learn more about what is being studied in the trial.Back to top
Why participate in a clinical trial?
According to the government’s web site, ClinicalTrials.gov, “participants in clinical trials can play a more active role in their health care, gain access to new research treatments before they are widely available, and help others by contributing to medical research.”1Back to top
What is a protocol?
A protocol is the study plan which the clinical trial is based. The plan is designed to protect the participants and to answer specific research questions. The protocol details who may participate in the trial, what will be done to participants, how long the study will be conducted, and everything that is required for the study.Back to top
What are the different types of clinical trials?
Five of the typical types of clinical trials are listed below. Some trials may be more than one type of trial. For example, a treatment trial may also have screening tests in it.

Treatment trials
These trials test new treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
Prevention trials
These trials look for better ways to prevent disease in people who have never had the disease or to prevent the disease from returning. Approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes (exercise, diet, etc).
Screening trials
These trials try to find a better way to detect certain disease or health conditions.
Quality of life trials
These trials explore ways to improve comfort and quality of life for individuals with chronic illness.
Nontherapeutic trials
These trials hope to answer specific research questions without using any type of treatment on participants. This may be accomplished by reviewing medical records, doing certain types of medical tests (such as a blood draw), and/or asking participants questions in a survey.

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What are the phases of clinical trials?
There are four phases of clinical trials, along with pilot studies. The trials at each phase have a different purpose and help scientists answer different questions.

Phase I trials
test a new drug or treatment in a small group of people (20-80) for the first time in order to evaluate the treatment’s safety, how much can be given safely, and to identify possible side effects from the treatment.
Phase II trials
typically test the treatment in a larger group of people (100-300) in order to see if the treatment is effective and to further evaluate the safety of the treatment.
Phase III trials
typically test the treatment in a large group of people (1,000 to 3,000) to confirm that the treatment is effective, monitor side effects, compare the treatment to other commonly used treatments, and collect additional information which should help allow safe use of the treatment. Patients are usually randomized between different treatments.
Phase IV trials
are usually post marketing studies that establish additional information about the treatment’s risks, benefits, and best use of the treatment.
Pilot studies
are usually Phase II studies but use a much smaller group of people (10-30) in order to determine if the study should be conducted on a much larger scale (enroll more subjects).

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What is a control or control group?
A control and/or control group are patients that typically do not receive the experimental drug or treatment. Instead, in many clinical trials, one group of patients receives the experimental drug(s) or experimental treatment(s) while the control group receives either the treatment currently used for for the illness or receives nothing at all. The information obtained from a control or control group is what is used to compare what happens to the people who receive the experimental drug or experimental treatment.Back to top
What is a placebo?
A placebo can be any form of a medication or a device which may look like the experimental drug or device that is being studied, except the placebo provides no treatment value and should not affect you in any way. Therefore, subjects assigned to receive placebo will not be exposed to any of the risks of the experimental treatment and will not receive any of the benefits of the experimental treatment.Back to top
What is randomization?
Randomization is the process where more than one type of treatment is available and the researcher wants to compare how people do receiving one treatment to the other. The treatment a study subject receives is determined by chance. Randomization is similar to flipping a coin or rolling dice. Neither you (the study participant) nor the researcher may select which group you are assigned to.Back to top

Protecting Clinical Trial Participants

The government, the Institutional Review Board (IRB), and the Informed Consent process

Is the government involved in all clinical trials?
No, the government is not involved in all clinical trials. However, according to our agreement with the government (called an Assurance), all clinical trials conducted at University of Florida / Shands will comply with federal regulations. These regulations include the Office of Human Research Protections (OHRP) Code of Federal Regulations 45 CFR 46 (also known as the “Common Rule”), the FDA’s rules and regulations applying to the use of investigational drugs or devices; and the FDA’s Good Clinical Practice guidelines.Back to top
How is the government involved in clinical trials at UF, Shands, or North Florida/South Georgia Veteran’s Health System (NFSGVHS)?
UF, Shands, North Florida/South Georgia Veteran’s Health System (NFSGVHS) each have an agreement with the government called an Assurance. These Assurances detail that clinical trials conducted by our faculty and staff, at our facilities, or with our patients will comply with appropriate federal regulations as stipulated in 45 CFR 46. FDA regulations (21 CFR) are applicable any time an investigational drug, device, or biologic are being tested.Back to top
How do UF, Shands, and the NFSGVHS protect research subjects?
All clinical trials conducted at UF, Shands, and the NFSGVHS must be reviewed and approved by an Institutional Review Board (IRB). Institutional Review Boards are required by the government in order to review research to insure the protection of research participants.Back to top
What is the Institutional Review Board (IRB) and how does it protect research participants?
IRB stands for Institutional Review Board and consists of a committee of physicians, statisticians, researchers, community advocates, and others. The United States government requires all research involving human subjects to be reviewed and approved by an IRB (1) prior to any subject participation and (2) periodically thereafter in order to protect the rights of human participants.Back to top
What is Informed Consent and how does it work?
Informed Consent is the process of (1) educating people about the key facts of a clinical trial and (2) allowing people to voluntarily decide whether or not to participate in the trial. There are typically three steps taken in order to inform potential participants about the study prior to taking part:

  1. Someone who is involved in and familiar with the trial explains the details of the study.
  2. Potential participants are provided with two copies of the IRB approved Informed Consent form. The potential participant should keep one of these forms for their records. The other form would be signed and returned to the investigator if the subject choose to take part in the trial.
  3. Potential participants should have an opportunity to ask questions about (a) the trial, (b) their condition/illness, and (c) alternatives to participating in the trial.

Informed Consent does not end once someone agrees to participate in a trial. According to federal regulations Informed Consent is an ongoing process which continues throughout the trial in order to insure that participants remain fully informed about the trial. For example, participants should be informed about any new information discovered during the trial, whether it is about the trial itself, the condition/illness being studied, or possible new alternatives to the trial.

The Informed Consent document is reviewed and approved by the IRB and describes the rights of study participants, details about the study (such as the study’s purpose, duration, required procedures, risks, benefits, alternatives, and key contacts). You are not required to sign the Informed Consent document unless you want to participate in the trial. The Informed Consent document is not a contract – participants may choose to withdraw from the trial at any time after agreeing to participate.

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Participating in a Clinical Trial

What You Should Ask and Know About a Particular Trial before Participating

What trial specific information should you know?
You should be provided with the following information

Title
What is the official title for the trial.
Phase
What phase trial is it? Different phases have different purposes and answer different questions.
Purpose
What is the trial studying, what does it hope to learn?
Eligibility criteria
Who may participate in the trial, and possibly who may not participate in the trial.
Trial duration
How long will the research be conducted?
Time commitment for subject
How much time does each research participant have to spend in order to be part of the trial.
Description
Explain what will be done to participants in this trial.

  • What kinds of tests and treatments are involved?
  • How might the trial affect my daily life?
  • Will I have to be admitted to the hospital if I participate in the trial?
  • Who will be in charge of your care?
  • How will you know if the treatment is working?
  • Who will pay for the treatment?
  • Will you be reimbursed for other expenses?
  • What will be done once you finish treatment?
  • Will results of the trial be provided to you?
Balanced description of risk vs. benefits
A realistic explanation of what may happen (both good and bad) to participants in the trial. Click here to learn more about risks vs. benefits.
Compensation for injury
How will you be compensated if you are injured as a result of participating in this trial.
Trial sponsors
Who is funding the trial.
Names of clinical investigators
Who is conducting the trial locally.
Contact info
Who you can contact with questions about the trial.
Alternatives to the trial
What other types of treatment are available if you choose not to participate in the trial.
Conflict of Interest
Do any of the researchers, the hospital, or the university have a conflict? Back to top
What is the significance of “risks vs. benefits”?
Participating in a clinical trial may expose people to known and/or unknown risks. Risks can consist of physical side effects from the study such as headache, nausea, hair loss, skin irritation, or a wide variety of other physical problems. Other types of risks could be financial or psychological. Some risks may occur immediately while participating in the trial while other risks may not occur until much later, sometimes years after participating in the trial. The risks should be detailed in the Informed Consent form and should be described to you by a knowledgeable researcher affiliated with the trial. If you choose to participate in a trial you should be notified of any changes in the risks associated with the study.The primary benefit of participating in a trial is that the knowledge obtained from the study may help other people in the future. Previous studies or other information may indicate that study subjects might benefit directly by participating in a research trial, but this can never be guaranteed.Thus, when participating in a clinical trial participants should compare the risks of participating to the benefits. Participants may decide that the potential benefits are not worth the risks.Back to top

What should you know about Conflict of Interest?
Sometimes people or institutions might benefit if the results of a clinical trial are positive. For example, a physician or the university might make money if they own the patents for a drug or device studied in the trial and the trial shows that the experimental treatment is effective. The University of Florida and the IRB try to ensure that any conflicts are not too large as to effect how well a clinical trial is conducted. The Informed Consent form potential subjects receive contains information about any conflicts in order to help potential subjects decide if they want to participate in the clinical trial.Back to top
Are there alternatives to the Clinical Trial?
First and foremost, no one is ever required to participate in a clinical trial. Participation in a clinical trial is voluntary, and potential subjects may always decide not to participate without it affecting their relationship with the researcher, other medical staff, the hospital, and/or the university. Most times there are alternative treatments that people can receive rather than participating in the clinical trial. Sometimes you can receive the same therapy without enrolling in the clinical trial. Other times the only way to receive a certain drug or device is by deciding to participate in the clinical trial.Back to top

1 http://www.clinicaltrials.gov/ct/gui/info/whatis