One of the fundamental goals of the Epilepsy Therapy Project is to help promote the discovery of new treatments for epilepsy and bringing those treatments to the patient sooner than later. One of the fundamental building blocks to achieve this is clinical research. Clinical research is a vital part of the Epilepsy Therapy Project’s mission, as research is the only way that we can progress to find new treatments and cures. The core element for all medical knowledge is derived from clinical trials. Clinical trials, in brief, are studies of possible new treatments or medications and are a segment of clinical research. Clinical research also can be used to look at other aspects of care, such as preventing disease, screening and or diagnosing health problems, and quality of life for people with chronic illnesses. Clinical trials are studies designed to find answers to specific health questions. Clinical trials often look at new medications or drugs, new combinations of drugs, new surgical procedures or devices, or new ways to use existing treatments.
However, before a new approach can be tested in humans, it must already have shown some benefit in laboratory testing, animal experiments, or in testing with a small group of volunteers. The goal of clinical trials is to determine if a new treatment both works and is safe. In fact, new medications cannot be sold in the United States until they have been through clinical trials. Therapies that prove effective during this research may go on to become approved and then widely available treatment options.
Who can participate in clinical trials?
Epilepsy researchers are often looking for individuals with chronic seizures with particular characteristics depending on the study. Some epilepsy trials are limited to people who haven’t started treatment, such as new onset epilepsy or new seizures. Many others focus on people where treatment has failed, the drug-resistant or refractory epilepsy patient group. Even in some instances, healthy individuals are needed for some trials. Determination of who gets enrolled in the trials also takes age, sex, and race into consideration when selecting individuals. The reason that this is done is because some clinical trials are designed to address just the young, some are designed to investigate particular groups like, young children, the elderly, women or men. The important point is that there are a lot of different options that can go into one’s selection for a trial.
Many people volunteer for a clinical trial because it is an opportunity to help researchers find a new way to fight disease. Treatment trials can provide access to new or experimental treatments that otherwise just wouldn’t be available because they are not approved. However, even though volunteering may help others in the future, there is no guarantee that the treatment will be effective for the patient at hand. Complicating this picture is that some of these trials use a placebo (a pill or liquid that looks like the treatment but has no active ingredients) as a comparison for the drug being tested. In a "blinded" trial, neither the participant or the doctor will know who gets the placebo and who gets active treatment. This is to prevent either party from having preconceived ideas about the outcome. Often, however, at the end of the blinded period, there is an open period which is usually much longer, during which everyone gets active treatment. The treating doctor performing the study will explain exactly how the study will run before a volunteer makes a decision about participating.
How do I find out about a clinical trial?
Sometimes the treating doctor may recommend a clinical trial for a given patient or an individual may actually find out about a trial and ask to participate. Oftentimes groups such as the Epilepsy Therapy Project, Epilepsy.com, and other advocacy organizations like the Epilepsy Foundation let their members know about clinical trials that are recruiting. In some instances, and particularly those in large cities, local news media like newspapers or radio may carry advertisements for clinical trials at nearby research centers. The National Institutes of Health posts listings of thousands of clinical trials online and Epilepsy.com has an entire section devoted to this topic. If an individual is interested in participating in a clinical trial it is important that they talk to the physician who is involved in the trial because this physician may be able to better provide information as to whether they have the right characteristics for the trial being considered and can also discuss the benefits and risks of participating. There may be some type of screening tests that are done prior to enrollment in order to make certain that the individual is appropriate for the trial.
It is important that you understand that you need to have as much information about the study and the treatment being investigated in order to make a well informed decision. Facilitators for these studies provide what is called an informed consent document, which is read and signed by the participant before they enter into the trial. This informed consent document contains information that one needs to know about the study before deciding whether to participate. Some of the information on an informed consent document includes:
This form needs to be explained in language that you can understand and no one should pressure an individual into signing up for a trial if there is some sense of hesitation. Signing this document means that one has agreed to participate in the trial, but it isn’t a contract, and you can leave the study at any time. If your child is enrolled a clinical trial you also go through this informed consent process together, but you have to give permission for your child to participate.
There are many questions that you should ask before participating in the trial. These questions should include the following:
Protocols: What will happen?
The map out for clinical trials follows an action plan that is laid out by the researcher called a protocol. The protocol explains what will be done in the study and outlines the following items:
To ensure the safety of people in the study the organization that sponsors the study, such as the medical center, clinic, or research institute must approve the protocol. There is a group mandated by the federal government called the Institutional Review Board (IRB) at each study site which reviews the protocol. The IRB includes people from the community as well as healthcare professionals. It reviews all protocols to make sure that participants are treated humanely and ethically. This committee also looks at issues, such as whether the likely benefit of the treatment is worth its risk. If the risks to the participants are found to be too great, the IRB can require changes in the protocol or reject the study. The IRB also checks to see if any of the researchers have significant conflicts of interest.
A clinical trial may also have another group called the Data Safety Monitoring Board overseeing it. This board is composed of people who aren’t directly involved in conducting the study. The Data Safety Monitoring Board can stop the study at any time to see if the treatment isn’t working or is causing too many side effects. A trial can also be halted by this board if the treatment is proven to be better than the standard treatment. It may be working so well that it should be made available to everyone outside of the clinical trials.
So what are the types of clinical trials?
Clinical research takes place in steps and each step has a different focus.
Phase 0 (Zero)
Phase 0 trials are a relatively new type of study. They are designed to weed out treatments that aren’t promising before they go on to what’s known as Phase I research. These trials are small, typically less than 10 people, and usually focus on how a drug acts in the body and how the body reacts to it.
This is typically the first step in testing in humans, and is often done in healthy volunteers rather than patients. At this point researchers study:
Earlier studies have often been done only in animals so the new treatment may pose some risk when first tried in humans. Researchers try to minimize these risks by starting with a very small dose and then increasing it only if there are no or few side effects. Phase I trials usually include a limited number of people, typically anywhere from 20 to 80 individuals. The number can vary depending on the goals of the research.
This step focuses on determining whether a new approach works as intended. For example, does this drug actually seem to stop seizures? Researchers also monitor participants for common, short term side effects that occur with the therapy. Some things aren’t yet known about the treatment so risks are still present. Several hundred people at many different centers may participate in a Phase II study.
If a treatment is shown promise in Phases I and II, it can move on to this phase. Participants are often randomly assigned, meaning neither the patient nor the doctor can choose which treatment is received, the experimental treatment or the standard treatment, or even the placebo. Randomization helps to avoid bias in the study and in doing so, improves the likelihood that the results are correct. Phase III trials provide more information about the safety of the experimental treatment and determines whether the standard or the experimental treatment has better outcome rates and fewer side effects. These trials usually include several hundred to several thousand people. Large trials make it easier to estimate what would happen if the experimental treatment were available to everyone with a condition. If a treatment performs well in a Phase III trial, the researchers may submit an application to the Food and Drug Administration (FDA), asking permission to make it available to the public. The FDA approval process generally takes about a year. The process is quicker for priority drugs, drugs that provide a significant advance in treatment for a life threatening condition. The FDA tries to rule on these within six months. In other cases it can take a few years in order to hear the final ruling.
In some cases research continues even after the FDA has approved a treatment. This research, sometimes called post-marketing research, tracks side effects in people receiving the newly approved treatment. Phase IV studies aren’t required for every treatment, but the FDA can ask a manufacturer to conduct a Phase IV trial to further evaluate long term safety and effectiveness. This has been done for epilepsy drugs ( i.e. Felbamate) Companies may also choose to do Phase IV research to compare their product with a competitor. Studies may also be done in new populations or seizure types that were not tested in the initial trials. Several hundred to several thousand people may take part in a Phase IV study.
Should I participate in a trial?
Researchers need volunteers for clinical trials, healthy people, as well as those at risk or diagnosed with epilepsy. By participating, you help researchers find new ways to stop epilepsy. Volunteering is also a way to access treatment that otherwise isn’t available. If you’re considering a clinical trial because you’ve been diagnosed with epilepsy, it is important to take time to learn about seizures and epilepsy and your options. It is essential that you talk with your physician, other healthcare providers, and discuss your options with those that are close to you.
How can Epilepsy Therapy Project help?
The goal of the Epilepsy Therapy Project is to bring new treatments for epilepsy to the patient with seizures in the safest and most efficient manner. The Epilepsy Therapy Project sponsors clinical trials, posts clinical trials, and has a section for both researchers as well as epilepsy patients so that they may best be educated as to what is available with regards to treatment. We are strong advocates of the clinical trial process and want to bring the best new treatments to the people who need them as soon as possible. Clinical epilepsy research is a way to accomplish this.
Joseph Sirven, M.D. and Jacqueline French, M.D.
Last Reviewed: 2/15/11
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