Clinical Trials: Another Option

The Epilepsy Therapy Project of the Epilepsy Foundation works to promote the discovery of new treatments for epilepsy and bring those treatments to the patient faster. One of the fundamental building blocks to achieve this is clinical research.

  • The core element for all medical knowledge is derived from clinical trials. These are studies of possible new treatments or medications and are a type of clinical research.
  • Clinical trials are designed to find answers to specific health questions. They often look at new medications or drugs, new combinations of drugs, new surgical procedures or devices, or new ways to use existing treatments
  • Clinical research also can look at other aspects of care, such as preventing disease, screening for and diagnosing health problems, and improving quality of life for people with chronic illnesses.

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. Clinical trials in larger groups of people are then done to see if a new treatment 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 for widespread use in people.

Who can participate in clinical trials?

  • Depending on what is being studied, people with a specific type of seizure or epilepsy may be sought.
  • Epilepsy researchers are often looking for individuals with chronic epilepsy, meaning that they have had epilepsy for a number of years.
  • 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 whose treatment hasn’t worked and who have refractory or drug resistant epilepsy.
  • In some studies, healthy people without epilepsy are needed.
  • Other factors are considered when determining who can participate in a trial, such as age, sex, race or if other medical conditions are present. For example, some clinical trials are designed to address young adults, while may be looking at 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.

What are some of the benefits or risks of joining a clinical trial?

Many people volunteer for a clinical trial because it is a chance to help researchers find a new way to fight disease. Treatment trials can provide access to new or experimental treatments that otherwise wouldn’t be available yet. There are other factors that people should consider.

  • Even though volunteering may help others in the future, there is no guarantee that the treatment will be effective for the people participating in the study.
  • The drug or device may not work well enough.
  • Some studies compare people taking a new drug with those who are given a placebo (a pill or liquid that looks like the treatment but has no active ingredients) as a comparison for the drug being tested. These studies are often "blinded", which means that the participant and the doctor do not know who gets the placebo and who gets active treatment until after the study is done. This is done to prevent people from having preconceived ideas about the outcome.
  • Often at the end of a blinded study, there may be an open period during which everyone gets active treatment.

If you are considering a clinical trial, make sure you learn exactly how the study will run and that you are comfortable with the benefits and risks.

How do I find out about a clinical trial?

Sometimes a doctor may recommend a clinical trial or you may learn about it on your own and ask to participate. Here are a few ways to learn about clinical trials in epilepsy:

If you are interested in participating in a clinical trial it is important to talk to the doctor and research team involved with the trial. You’ll need to learn about the study and whether you could be eligible for it. You may need to have some screening tests first to make sure that you are appropriate for the study.

What is informed consent for research?

  • Informed consent is the process that’s used to make sure that a person has as much information about the study and the treatment being investigated to make a well informed decision.
  • The researchers must provide an informed consent document, which is read and signed by the participant before they enter into the trial. Some of the information on an informed consent document includes:
    • What is going to happen during the trial, including the test you may be given
    • Known risks and benefits of the experimental treatment
    • How long you will be expected to participate in the trial
    • Other treatments that might help you instead of the treatment being tested
    • Who to call with questions
  • This form needs to be explained in language that you can understand. You should also have plenty of time to ask questions and make up your own mind. No one should ever free pressured to take part in a research trial.
  • Signing this document means that you have agreed to participate in the trial, but it isn’t a contract. You can leave the study at any time.
  • If your child is enrolled in a clinical trial the parent(s) and child go through this informed consent process together. The parent or guardian has to give permission for their child to participate.

What questions should I ask before deciding to be in a research study?

There are many questions that you should ask before participating in a research study or clinical trial. Some of these include the following:

  • Who is in charge of the study?
  • Do the people running the study have a conflict of interest in the outcome?
  • What is being found out?
  • What is expected of me?
  • Is it possible that I might receive a placebo?
  • What benefits or risks can I expect?
  • How long will I be in the study?
  • What happens if my condition gets worse during the study?
  • Can I see my own doctor during the study?
  • Do I need to pay for any part of this study?
  • Who pays if I am injured in the study?
  • How will participation affect my life?
  • What happens at the end of the study?
  • Will I ever be told the results of the study and when?
  • Who will know that I am participating?

What will happen during the study?

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:

  • Who may participate
  • How many people take part
  • What will the treatment plan entail
  • The type and frequency of tests
  • How will the results be measured
  • The reasons the study may be stopped
  • The situation in which researchers may stop giving the treatment to patients
  • Known or possible side effects
  • Possible benefits of the treatment

How is the safety of clinical trials monitored?  

  • 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.
  • 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 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. This board can also stop a trial 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 further steps. 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.
  • Phase I: This is typically the first step of testing in humans, and is often done in healthy volunteers rather than patients. Usually a limited number of people are needed for this type of study, typically 20 to 80 people. At this point researchers study:
    • How much of the treatment to give?
    • When should it be given and how often?
    • What is the highest dose that can be given safely?
    • How the body reacts to the treatment?
    • Any harmful side effects?  
  • Phase II: The goal of this step is to find out whether a new approach works as intended. For example, does this drug actually seem to stop seizures? Researchers also monitor people 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.
  • Phase III: If a treatment look promising in the first two phases, it can move on to this next phase. Participants are often randomly assigned to either the experimental treatment or a standard treatment or a placebo (inactive drug). Neither the patient nor the doctor can choose which treatment is received. Randomization helps to avoid bias in the study and improves the chance that the results are correct.
    • Phase III trials provide more information about the safety of an experimental treatment and whether the standard or the experimental treatment works better and has 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.
  • Phase IV:  Research may also continue 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 collect information on long term safety and effectiveness.
    • This has been done for some epilepsy drugs ( i.e. Felbamate) when safety concerns were found after the drug was approved.
    • 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, people without epilepsy, 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 the Epilepsy Foundation 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 people with epilepsy 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.

Authored by: Joseph I Sirven, MD | Jacqueline French, MD
Reviewed by: Patricia O. Shafer RN, MN | Joseph I Sirven, MD on 11/2013
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