Epilepsy clinical trials study possible interventions for seizure prevention. Interventions might include medications, procedures, or behavioral changes.

Researchers often conduct lab studies to establish risks and benefits before moving to human trials. Those with epilepsy participate in clinical trials to receive cutting-edge treatment and contribute to valuable research.

This article reviews epilepsy clinical trials, what they are, how they work, types, benefits, risks, participation requirements, and frequently asked questions when considering participation.

What Are Epilepsy Clinical Trials?

Epilepsy clinical trials study new possible interventions to stop seizures. They might center around:

  • Medications with fewer side effects
  • More effective medications
  • Treating drug-resistant epilepsy
  • GABA-A (gamma-aminobutyric acid receptor agonists) (stimulate melatonin receptors)
  • Dietary changes (such as the ketogenic diet)
  • Gene therapy
  • Immunosuppressive therapy
  • Vagus nerve stimulation
  • Neuroinflammation-targeting treatments (treats inflammation in the brain or spinal cord)

While there is no cure for epilepsy, trials have led to more specific and effective treatments with fewer side effects. For example, Epidiolex (cannabidiol) is a newer medication that prevents seizures for some with previously drug-resistant epilepsy

How They Work

Various healthcare, academic, and pharmaceutical institutions fund clinical trials. Locations include research centers, clinics, hospitals, and universities.

Researchers begin with a protocol that maps out the study design. The design includes eligible participants, sample size, and duration.

A safety board approves the protocol, and the research team begins the clinical trial. This team includes a principal investigator, healthcare providers, and social workers. Human volunteers review the protocol, give consent, and become part of the team.

Types of Clinical Trials

Clinical trials study experimental medications, devices, procedures, or therapies. Their goals vary and may center around the following:

  • Prevention: Focus on keeping seizures or epilepsy from occurring
  • Screening: Find ways to detect disease sooner
  • Diagnostics: Determine better, less invasive ways to diagnose
  • Treatment: Test interventions to see if they are more effective than current epilepsy treatment
  • Quality of life: Look for better ways of supporting those with a severe or debilitating epilepsy

Most people are familiar with treatment or interventional clinical trials. Depending on their study design, participants may get the new intervention, traditional treatment, no treatment, or a placebo.

A placebo is a substance that doesn’t have a therapeutic effect. However, some protocols stipulate that every participant receives the treatment once the trial concludes.

Studies that utilize placebos randomly assign participants the treatment or the placebo to decrease the risk of bias. There are two types of placebo studies:

  • Single-masked: Only the research team knows who is getting the treatment.
  • Double-blind: Neither the participant nor the research team knows who receives the treatment or placebo.

Informed Consent

Informed consent ensures participants have ample information before enrolling in a study. Researchers provide documents or question-and-answer sessions about expectations, timelines, risks, benefits, and alternatives.

Once participants or guardians feel comfortable, they sign an informed consent document. This document is not a contract; participants can withdraw at any time.

Food and Drug Administration (FDA) approval is necessary to prescribe medications in the United States. The FDA requires medication clinical trials to go through the following phases before approval.

  • Early phase 1: Includes fewer than 15 participants and focuses on safety and effectiveness
  • Phase 1: Involves 20 to 100 people and focuses on safety, effectiveness, adverse effects, and the behavior of the medication
  • Phase 2: Goals that are similar to phase one but with a more broad population, typically several hundred people
  • Phase 3: Includes 300 to 3,000 people, and if it goes well, researchers apply for FDA approval
  • Phase 4: Post-approval phase that involves over 1,000 people and may include new populations, such as children

Benefits and Risks of Joining a Clinical Trial

Clinical trials are essential tools for medical advancement. But it’s important to understand the risks and benefits before volunteering for one.

Benefits include:

  • A potential new treatment is available to you before it’s released to the general public.
  • You receive more frequent healthcare checkups.
  • Treatment is from expert medical professionals in their field.
  • Increased access to resources is available.
  • Treatment is free.
  • You will be contributing to valuable research.

Risks include:

  • Side effects of new interventions are not always known.
  • It can take time, and you may need to travel.
  • There is no guarantee the intervention will work.
  • There is no guarantee you are getting the treatment.

Participating In a Clinical Trial

Clinical trial protocols outline inclusion criteria, expectations, treatment regimens, and the length of the study. If they aren’t outlined, don’t be shy about asking the researchers. They want you to fully understand and be comfortable before joining.

Who Can Join?

Clinical trial protocols typically include eligibility criteria. For example, some require healthy volunteers, while others only accept those with epilepsy. Eligibility criteria may also involve:

  • Age
  • Gender
  • Health conditions
  • Treatment or medication history
  • Drug-resistant epilepsy
  • Has new-onset epilepsy or long-term epilepsy
  • Specific types of epilepsy or seizures

Where to Find Trials

You can ask your healthcare provider, local hospitals, community clinics, or universities if they know of any current clinical trials in your area.

The National Library of Medicine has a searchable clinical trial database. You can also go directly to its epilepsy clinical trials database.

Once you find a study you are interested in, it will provide you with inclusion criteria. The advanced search feature also allows you to sort trials by the following criteria:

  • Study type
  • Previous study results
  • Status (if they are recruiting)
  • Age
  • Gender
  • Intervention
  • Location
  • Phase
  • Funding
  • Start date
  • Estimated completion date


Researchers often begin their study with preclinical laboratory (in vitro) studies. This provides baseline information regarding safety before moving to human clinical trials.

In the United States, federal laws have strict rules to protect your safety, rights, health, and privacy. These laws include utilizing an institutional review board (IRB). This board must approve the protocol before the trial begins.


What Is an IRB?

An IRB comprises doctors, researchers, and nonmedical community members. They review the protocol to ensure it’s ethical and safe. IRB members assess risks and benefits and can ask for protocol changes. They also review informed consent documents and monitor for conflicts of interest.

Some studies also have a data monitoring board that includes people who are not directly involved in the study. They can stop the study anytime if they are concerned about safety or efficacy.

The Office of Human Subjects Research Protection and the FDA can also determine whether a study adequately protects participants.

Questions to Ask Before Joining

The following list of frequently asked questions may help you decide on joining a clinical trial:

  • What is the trial studying?
  • Has it already been tested?
  • Who’s sponsoring the trial?
  • How will you determine who gets the intervention?
  • Is it possible that I might receive a placebo?
  • What are the benefits, risks, and possible side effects?
  • How does the new intervention compare with my current epilepsy treatment?
  • What are your expectations and requirements for me?
  • Who pays for my participation?
  • Does the study reimburse me for any expenses?
  • How long is the trial?
  • What happens at the end of the study?
  • If the intervention works, will I be able to keep taking it?
  • What happens if my seizures get worse during the trial?
  • Can I see my own healthcare providers during the trial?
  • How is my privacy protected?

Discussing Clinical Trials With Your Healthcare Provider

Speak with your healthcare provider(s) before joining a clinical trial.  They may want to ask the research team questions about the possible intervention. This helps them know if they need to adjust any of your treatments and how to monitor you. There may also be screening tests they need to order before you begin the trial.


Epilepsy clinical trials study new possible interventions, including medications, procedures, and behavioral changes. While there is no cure for epilepsy, previous trials have led to more specific and effective treatments with fewer side effects.

Participants join clinical trials to gain access to preapproved, new treatments and to contribute to valuable epilepsy research. If you want to join one, check their protocol to see if you meet the eligibility criteria.

In the United States, federal laws have strict rules to protect participants’ safety, rights, health, and privacy. These processes include informed consent, IRB boards, data monitoring boards, and federal agency oversight.


  • Is there a cure for epilepsy?

    While there is no cure for epilepsy, there are treatments that reduce or eliminate seizures with minimal side effects. Treatment works best when the proper medication is prescribed based on a person’s specific epilepsy syndrome, seizure type, and genetic mutations.

  • Have there been any recent advancements in epilepsy treatment?

    Yes, there have been incredible advancements in epilepsy treatment. For example, Epidiolex (cannabidiol or CBD) is a new medication that treats forms of drug-resistant epilepsy. One new clinical trial involves the Targeted Augmentation of Nuclear Gene Output (TANGO), a type of gene therapy.


  • How do you know if you’d be a good fit for an epilepsy clinical trial?

    Clinical trials have a protocol that includes eligibility criteria. The National Library of Medicine epilepsy clinical trials database includes this information for most studies.


Source: verywellhealth.com, Brandi Jones MSN-ED RN-BC, Brigid Dwyer MD