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What is a clinical research trial?
A clinical research trial is a scientific study that has been carefully designed to answer a very specific medical question. Clinical research trials are often referred to as “drug studies.” Some studies test medications that are currently unavailable for treatment in the United States (referred to as “investigational new drugs”), whereas other studies examine medications that your doctor can prescribe today. All new drugs are tested first in animals, then normal human volunteers, and finally in patients with a specific disease, such as asthma.
Who conducts clinical trials?
The company that develops a new drug is called the “sponsor.” The sponsor generates the specific question to be answered and designs a set of guidelines (or “protocol”) that personnel conducting the study must follow.
Most clinical trials require large numbers of patients (usually several hundred) to correctly answer the research question. Since sponsors do not have direct access to patients, they typically ask medical practices across the U.S. (or world) for help in recruiting patients who may want to help in the study. These practices, such as ours, are known as “investigative sites,” and serve as the locations where the clinical research trial is actually conducted.
Who is eligible to participate?
Because a clinical research trial is designed to answer a specific medical question, sponsors request that only patients with a specific condition or degree of symptoms participate. These are known as “inclusion criteria.” Protocols will also specify that patients not have coexisting medical problems that could make the data difficult to analyze. These are known as “exclusion criteria.” Inclusion and exclusion criteria vary from study to study, so that a patient who might not qualify for one study would be an excellent candidate for another. The doctor or study nurse is the best person to determine whether you will be eligible to participate.
What is it like to participate in a clinical research trial?
Participating in a clinical research trial is a partnership or commitment between you and the study team. You will typically be asked to keep a written or electronic record of your symptoms on a daily basis. You will need to record when you take study and other medications. Many asthma trials require you to perform breathing tests at home. On occasion, you will need to come to our office for scheduled study visits for physical exams, breathing tests, labwork and to complete various questionnaires. In short, you will receive a great deal of personalized medical attention, while at the same time learning a fair amount about your disease.
What risks are involved? Is it safe
All medications have various side effects and risks. Many of these side effects are not unexpected (i.e. upset stomach with aspirin), although some occur for no apparent reason. The same holds true for investigational medications. It is important to remember that study medications are tested first in animals and then in normal human volunteers, before being approved for testing in patients with a specific disease. Any side effects identified during these studies are reported to each investigative site before the trial begins, and it is your right to know about these side effects.
Because the clinical experience with an investigational medication by definition is limited, several measures are taken to ensure your safety:
- An independent group (called an institutional review board, or IRB) makes sure that your rights and welfare, as a human subject participating in medical research, are protected. The IRB approves the protocol, the investigative site, and the consent form that you will be asked to read and sign before participating. Investigative sites have an obligation to inform the IRB of any serious adverse events which occur during the trial (whether felt related to the study medication or not), and the IRB has the authority to immediately stop research activities if they have concerns regarding patient safety.
- To minimize the potential for unexpected side effects, most protocols require close monitoring of study patients by serial physical exams and lab work (such as blood tests, EKGs, etc.). Patients are not allowed to continue in a study if any worrisome changes develop during the course of the trial.
- The sponsor alerts each investigative site of serious adverse events that occur during the trial. This makes us look out for potential problems in our group of patients. Again, your safety is our primary concern.
What are the benefits? Why should I participate?
By participating in a clinical research trial, you will receive a great deal of personalized medical attention, including physical exams and lab work. You may receive a new medication that others with your condition are unable to receive today, and which has potential to improve your health. Most studies, however, do not allow you to continue to receive an investigational medication after you finish the trial. You will probably learn a great deal about your disease, and at the same time will be helping others by taking part in scientific research. Once enrolled, study-related care (including physical exams, lab work, breathing tests and study medications) is provided free of charge, and study patients, in addition, are compensated for their time and travel.
What will be expected of me?
Your decision to participate should not be taken lightly, nor should you do this simply out of curiosity. Remember, the data that you generate will be combined with that of others in the study to determine whether or not the investigational medication may be made available for future treatment in the U.S.
You will need to keep your appointments on time, follow instructions and take medications exactly as directed. At each visit, you will need to inform us of any new symptoms that develop, when they started, when they stopped, how severe they were, and whether they required the use of any other medications. Some protocols have very specific guidelines on what medications can and cannot be used with study medication. For example, an OTC cold medicine could be used for a respiratory infection in some asthma protocols, but not others.
What can I expect from the study team?
We will give you complete information about the study, including purpose, duration, number of visits, expected time commitment, study procedures, permitted medications, previously reported side effects, risks, benefits, and compensation. We will also ask you to carefully read, ask questions about, and sign an informed consent form that explains many of these elements, and contains additional information regarding alternative treatments, confidentiality, who to contact for questions, voluntary participation, circumstances that may result in termination of participation, and compensation for injury. It is our goal that you are fully informed as to what to expect from the first time you come into our office until your last study visit.
Once I start a study, am I obligated to complete it?
Your participation in a clinical research trial is entirely voluntary. You will not be penalized or lose benefits to which you are otherwise entitled if you refuse to participate or withdraw from active participation.
How will participating affect my relationship with other doctors?
You are encouraged to continue to see regular physicians involved in your ongoing care; participation in a clinical trial should in no way alter these relationships. Unless you object, it is our policy to send your doctor a brief letter informing him/her of your participation once you are enrolled.
The fact that you are on study medication should not affect the management of any other medical problems that develop during the course of a trial. If you develop an illness, we encourage you to contact us, so that we can tell you whether we can evaluate your problem, or whether it is best dealt with by your regular doctor. We do ask, however, that no medical personnel other than the study nurse or one of our doctors make decisions regarding your study medication.