What is a clinical study?
A clinical study, also called a clinical trial, is an organized experiment performed on human participants to answer a specific scientific question. Clinical studies are done to determine the best way to treat, diagnose or prevent a disease. A clinical study could test the effectiveness of a new drug or medical device such as an artificial hip, see how well a new medical test or surgical technique works, study feelings or behaviors, or simply gather other types of information.
The purpose of a clinical study is to answer questions about safety and effectiveness. They help scientists determine whether we are using the best treatments or to help the U.S. Food and Drug Administration (FDA) determine whether or not to approve a treatment for general use. The information scientists learn from clinical studies helps to improve the medical care for future patients.
What are the different types of clinical trials?
Treatment trials test experimental treatments, new combinations of drugs, or new approaches to surgery or radiation therapy.
Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vitamins, vaccines, minerals, or lifestyle changes.
Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular disease or condition.
Screening trials test the best way to detect certain diseases or health conditions.
Quality of life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.
What are the phases of a clinical trial?
A pilot study is a first look to test an idea. It usually involves one or a very small number of participants. Because it is so early in the development of the idea, there is very little specific information for the participants and very careful observations are required. These studies are designed to be completed in a short period of time.
Phase I Study
Focus is on safety in humans, and it usually lasts from 6 to 12 months. The study involves initial administration of an investigational drug to a small number of participants to determine the safe dose range for the drug. Observations are made regarding how the drug is absorbed, metabolized, eliminated and to test for obvious side-effects. Some evidence of the drug’ potential effectiveness may also be obtained. Phase I studies may be performed on healthy volunteers, or on participants with the specific disease the drug is intended to treat.
Phase II Study
The clinical administration of a new drug to a limited number of participants for a short period of time to determine the drug’ safety and benefit in treating or preventing disease.
Phase III Study
The administration of a new drug to a large number of participants in different clinical settings to determine its benefits and safety. In Phase III studies, a drug is used as it would be when ready to be marketed. When these studies are completed and the sponsor can demonstrate that the drug is safe and effective under specific conditions, the sponsor applies to the Food and Drug Administration (FDA) for clearance to market the drug to the public.
Phase IV Study
Clinical studies for determination of after-effects and secondary results. The study is conducted after the FDA has cleared the drug for marketing. It is initiated by the producer of the drug to gain more information about its safety and benefits.
Why are clinical trials important?
Advances in medicine and science result from new ideas or approaches developed through research. However, any new treatment can carry risks as well as benefits. Clinical studies help identify how safe and effective a treatment is for its intended use. In the United States, new drugs or devices must be shown to be safe and effective in clinical studies of a certain number of participants before the Food and Drug Administration (FDA) can approve them for use. Until then, they are considered investigational.
For existing treatments, clinical studies can find out which might be the best for certain types of patients, which might be the most cost-effective, or which might be easier to give. Studies also can help confirm or refute common medical beliefs. For example, aspirin’s protective effects against heart attack have been proven time and again. On the other hand, although “everybody knows” that a diet high in fiber can prevent colon and rectal cancer, a recent study has shown that this conventional wisdom may be wrong.
Who can participate in a clinical trial?
All clinical trials have guidelines about who can participate. Using inclusion/exclusion criteria is an important principle of medical research that helps to produce reliable results. The factors that allow someone to participate in a clinical trial are called “inclusion criteria” and those that rule out someone from participating are called “exclusion criteria.” These criteria are based on such factors as age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. Before joining a clinical trial, a participant must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help ensure that researchers will be able to answer the questions they plan
What Should I Know Before I Participate?
You should find out as much as possible before agreeing to participate in a clinical trial. You should talk with the doctors and nurses who are doing the trial, as well as with your physician, family and friends before making a decision. You also should seek to understand the credentials and experience of the individuals and the facility involved in conducting the study.
Here are some things to consider:
- What is the purpose of the study?
- What are the risks?
- How might I benefit?
- Who is going to be in the study?
- Why do researchers think the treatment being tested may be effective? Has it been tested before?
- What do I have to do to participate?
- What kinds of tests and treatments are involved?
- How do the possible risks, side effects, and benefits in the study compare with my current treatment?
- How might this trial affect my daily life?
- How long will the trial last?
- Will hospitalization be required?
- Will participation cost me anything, or will I be reimbursed?
- Is there any long-term follow up?
- How will I know that the treatment is working? Will results of the trials be provided to me?
- Will I get study results?
- Who will be in charge of my care?
What happens after I complete the registration process in the Research Participant Registry?
Once you are registered in the Research Participant Registry (RPR), there is the possibility that you may or may not qualify for any studies at that time; however, you will receive emails pertaining to research studies as they become available. It may be someone from WU or someone from one of the ICTS Institutions that contacts you
What kind of preparation should a potential participant make for the meeting with the research coordinator or doctor?
- Plan ahead and write down possible questions to ask
- Ask a friend or relative to come along for support and to hear the responses to the questions
- Bring a device to record the discussion to replay later
What happens after I am accepted as a participant?
After you agree to participate in a clinical study there are several steps designed to protect your health and rights as a research participant. The steps are as follows:
The Screening Process
You will meet the study coordinator and the doctor who will be keeping track of your progress throughout the study. The study will be explained to you including the potential benefits as well as the known risks. The participant signs a written version of this information prior to any study-related procedures being performed. This witnessed document is called an informed consent. Your medical history and current medication information is documented. The doctor will give you a physical exam and other specialized tests that might be required. If you qualify for the study you will receive the study medication and a follow-up appointment will be scheduled. Financial compensation for your time and travel may be available depending on the study. You will not be charged for medical care relating to your study participation.
During the study you will come in for appointments during which you will be asked questions as to how you are doing and about any other medications you have taken since your last visit. Depending on the study, additional tests may be required during the study process. You may have additional physical exams conducted by the study doctor. Your compliance in taking the study medication is assessed and the next set of study medications will be given. Your next appointment will be scheduled.
After the Study
When the study is completed, the study coordinator will once again assess how you are doing and check your compliance during the last segment of the study. The doctor may meet with you for one last physical exam and discuss with you the appropriate recommendations for follow-up care. The study coordinator may also discuss with you other clinical trials for which you may be qualified and tell you when you would be eligible for enrollment.
What happens during a clinical trial?
The clinical trial process depends on the kind of trial being conducted. The clinical trial team includes doctors and nurses as well as social workers and other health care professionals. They check the health of the participant at the beginning of the trial, give specific instructions for participating in the trial, monitor the participant carefully during the trial, and stay in touch after the trial is completed. Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of trials, the participant works with a research team. Clinical trial participation is most successful when the protocol is carefully followed and there is frequent contact with the research staff.
What are side effects and adverse reactions?
Side effects are any undesired actions or effects of drug or treatment. Negative or adverse effects may include headache, nausea, hair loss, skin irritation, or other physical problems. Experimental treatments must be evaluated for both immediate and long-term side effects.
How is the safety of the participant protected?
The ethical and legal codes that govern medical practice also apply to clinical trials. In addition, most clinical research is federally regulated with built-in safeguards to protect the participants. The trial follows a carefully controlled protocol, a study plan which details what researchers will do in the study. As a clinical trial progresses, researchers report the results of the trial at scientific meetings, to medical journals, and to various government agencies. Individual participants’ names will remain secret and will not be mentioned in these reports.
The participant’s rights and safety are protected in two important ways. First, any physician awarded a research grant by a private company or the National Institutes of Health must obtain approval from an Institutional Review Board (IRB) before conducting the study. An IRB is an independent committee of physicians, statisticians, community advocates, and others that ensure that a clinical trial is ethical, protects participant’s rights and does not present undue or unnecessary risk to the participant. Consequently, every clinical trial at Washington University must be approved and monitored by the Washington University IRB. Each ICTS Institution must also receive approval and be monitored by their IRB of record.
Second, anyone participating in a clinical trial in the United States is required to sign an “informed consent” form. This form details the nature of the study, the risks involved, and what may happen to a participant in the study. The informed consent tells participants that they have a right to leave the study at any time.
What is informed consent?
Informed consent is the process of learning the key facts about a clinical trial before deciding whether or not to participate. It is also a continuing process throughout the study to provide information for participants. To help someone decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the participant’s native language is not English, translation assistance can be provided. Then the research team provides an informed consent document that includes details about the study, such as its purpose, duration, required procedures, and key contacts. Risks and potential benefits are explained in the informed consent document. The participant then decides whether or not to sign the document. Informed consent is not a contract, and the participant may withdraw from the trial at any time.
What if I read something on the consent form that I don’t like?
Although previously you might have talked to the study doctor or study nurse, you might read the consent form and find out something about the clinical trial that you didn’t expect. Perhaps it never occurred to you to ask about the risks of a certain procedure, or a placebo period. Maybe you didn’t know that there would be an overnight study visit, and maybe any one of these details is enough for you to decide that you don’t want to join after all. You can thank the study staff for their time and effort, but at the same time politely decline to sign the consent form and leave the research center. There will be no hard feelings.
Does anyone monitor clinical trials?
The federal government has in-depth regulations regarding the protection of research study volunteers. The Food and Drug Administration (FDA) is a federal agency with which large drug companies must comply in order to have their drugs FDA approved and licensed for sale to the general public.
According to the FDA, any drug must pass rigorous safety tests in animals before it can be approved for clinical trials. Once the clinical trial begins, the FDA closely monitors its progress. In addition to approval by the FDA, every clinical trial requires approval and monitoring by Institutional Review Board (IRB). The IRB is a committee made up of doctors and community representatives. The purpose of the IRB is to review research and determine if the rights and welfare of research volunteers are adequately protected.
The study doctor is directly responsible for your safety. He or she will be watching for any abnormalities in test results.
Does a participant continue to work with a primary health care provider while in a trial?
Yes. Most clinical trials provide short-term treatments related to a designated illness or condition, but do not provide extended or complete primary health care. In addition, by having the health care provider work with the research team, the participant can ensure that other medications or treatments will not conflict with the protocol.
Can a participant leave a clinical trial after it has begun?
Yes. A participant can leave a clinical trial, at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the study.
Where do the ideas for trials come from?
Ideas for clinical trials usually come from researchers. After researchers test new therapies or procedures in the laboratory and in animal studies, the experimental treatments with the most promising laboratory results are moved into clinical trials. During a trial, more and more information is gained about an experimental treatment, its risks and how well it may or may not work.
Who sponsors clinical trials?
Clinical trials are sponsored or funded by a variety of organizations. Trials can take place in a variety of locations, such as hospitals, universities, doctors’ offices, or community clinics. Washington University sponsors certain clinical studies initiated by the physicians as part of its ongoing commitment to clinical research. Additional funding may come from the federal government (via the National Institute of Health), voluntary health agencies such as the National Multiple Sclerosis Society, private industry (via pharmaceutical and biotech companies), or from philanthropic private donations to Washington University that are earmarked for research. According to Washington University policies, physicians cannot be paid for conducting clinical studies. Instead, they are allowed the time required to give proper attention to each participant of a clinical study. The same applies to any ICTS Affiliated Institution.
What is a protocol?
A protocol is a study plan on which all clinical trials are based. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.
What is a placebo?
A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the experimental treatment’s effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or experimental treatment.
What is a control or control group?
A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of participants will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
How can I be sure that I am assigned to the study drug that I want?
Well, you actually can’t. The study group you are assigned to decides randomly, like flipping a coin or drawing a card. Often, a computer program randomizes participants. The study doctor has no influence as to which study group you are assigned within a given clinical trial.
Would I be the first person ever to take the experimental drug?
In order for a drug to be approved by the Food and Drug Administration, it must go through different phases of drug development. You can find out what phase your clinical trial is in by asking the study doctor or any member of the clinical trial staff. There are either Phase I, II, III, or IV clinical trials.
Will it cost me anything to participate? Will I be paid?
In general, there is no cost to volunteers or their insurance agencies for any research related costs. Often insurance agencies are billed for procedures that would be done regardless of participation in the research study. Some studies offer to pay the volunteer for their participation. This is usually a modest amount meant to cover travel and parking expenses and any inconvenience the study might cause you. For example, studies that require overnight visits to the research center offer a more generous remuneration than other studies. Compensation can still be given for study visits performed, even if an individual does not complete the trial. If during any calendar year, the amount of compensation for your participation in clinical trials exceeds $600.00, you will receive a 1099 from Washington University and be required to pay taxes on the total.
Will I know the name of the medication or medical device I may be taking or using?
Yes. You will be informed of the name of the medication or medical device involved in the study. This information can be found on the informed consent form.
How much time is involved?
Each study is designed differently. Most of the studies do involve a pre-study physical exam appointment, study involvement time, and follow-up visits. The informed consent will tell you the length of time involved. You always reserve the right to drop out of any study, at any time, for any reason.
What is a screening physical?
To become qualified for a study, certain study requirements must be met. Each study requires a screening physical that may include collecting blood and urine samples. You will be asked questions concerning your health history. You will be asked to read and sign the informed consent. This is also a good opportunity for you to ask additional questions about the study and the informed consent.
Does every enrolling clinical trial at Washington University use the Research Participant Registry to recruit participants?
No. It is our vision to make sure that all of the clinical trial teams at Washington University are aware of the RPR, but some research teams do not opt to use the RPR database. There are many reasons for that. However, Volunteer for Health (VFH) staff can usually get you in contact with the individuals with certain ongoing studies or areas of interest, once you are registered.
How will my privacy be protected?
Your personal health information is kept in the strictest confidence. It will only be seen by Volunteer for Health staff and relevant matched study teams. All of the individuals who have access to your personal health information will be fully trained to comply with federal health privacy laws — namely, the Health Insurance Portability and Accountability Act (HIPAA) — and in human subjects protection practices. They will maintain the utmost protection, security and confidentiality. Your personal health information will be shared with no one else. Contact information shared with an ICTS institution will be provided in a password-protected document.
Will I receive care at Washington University School of Medicine or Barnes-Jewish Hospital?
You will most likely receive care at Washington University or Barnes Jewish Hospital. Investigators sometimes have satellite offices where research is conducted near Washington University and Barnes-Jewish Hospital, but the majority of the clinical studies are performed at those two locations. However, if you are contacted by an ICTS-Affiliated Institution the study will likely be conducted at their site(s).
Why does the General Health Questionnaire ask about my race?
We ask about race or ethnicity for demographic purposes. At any time, we like to query our database to see if the database is an accurate representation of the local population. This information will in no way exclude you from matching potential clinical trials that are seeking participants.
What if I need to update my information in the Research Participant Registry?
Call or email Volunteer for Health (VFH) at firstname.lastname@example.org or 314-362-1000.
What if I no longer want to be in the Research Participant Registry?
You can request to be taken out of the RPR at any time. Just contact us by e-mail at email@example.com or call us at 314-362-1000 to let us know of your desire to be removed.
Will I receive information about the final results of the clinical trial in which I participate?
You can ask the study team if they will inform you of the final results.
Who should I contact if I have any questions?
You may contact Volunteer for Health (VFH) at 314-362-1000 with any questions or email us at firstname.lastname@example.org.