Clinical trials, also known as research or clinical studies, are essential for finding new and effective treatments for epilepsy.¹ People living with epilepsy can volunteer to join a trial and contribute to clinical research aimed at finding effective treatments and gaining a better understanding of their illness. Those who participate can help pave the way for more treatment options for epilepsy to become available in the future.

See if You May Be Eligible for a Clinical Trial

If you’re interested in possibly joining a clinical trial for epilepsy, here are six things you should know.

1. New Drugs Are Tested Before Clinical Trials Start

Before a clinical trial is designed, a potential new treatment is studied in the laboratory. Preclinical studies often take years. Researchers study cells, computer models, and results of animal tests to help determine the preliminary effectiveness and safety of a potential drug.² If preclinical research does not find unreasonable safety concerns, research teams will design a clinical protocol (research plan).³

Clinical trials are carefully designed around four phases that test the safety and effectiveness of a new drug. When a trial advances to the next phase, the researchers recruit more people and collect more detailed data on the therapy.⁴

2. Your Safety Is Top Priority During a Clinical Trial

Each country has specific rules for approving clinical trial designs. In the United States, a clinical trial plan must be reviewed by the U.S. Food and Drug Administration (FDA) to make sure that participants will be protected from unreasonable risk.³

The FDA also requires clinical trials to be reviewed by an institutional review board, which oversees the well-being and rights of volunteers. Participants’ safety is rigorously monitored and reviewed during each phase. If a drug is found to have unreasonable side effects, the trial will be suspended.⁵

3. You’ll Be Fully Informed About Details Before Joining

Before enrolling in a clinical trial, you’ll receive detailed information about the trial, possible risks, and what will happen during the trial. Volunteers must be given adequate time to consider research participation and ask questions.⁶

Called “informed consent,” this process fosters open communication between researchers and participants. You’ll be given time to discuss a clinical trial with your health care providers and family members. You’ll also have a chance to discuss all aspects of the trial, including the time required, whether travel costs are reimbursed, and any out-of-pocket costs.⁶

4. ‘Placebo’ Doesn’t Mean ‘No Treatment’

If you’re enrolling in a clinical trial because you’re interested in helping to evaluate a potential new drug, it’s important to understand that you might receive a placebo. Placebos contain no actual medication and don’t pose a risk, but if you’re given a placebo, you won’t be getting the investigational drug.

Clinical trials often include a randomized, double-blind placebo control group. This means that a randomly chosen group will receive a placebo (no active drug) instead of the investigational drug.¹ Being in the placebo group doesn’t necessarily mean that you’re not receiving any treatment — depending on the trial, you may receive a placebo in addition to the standard treatment for your condition.⁷ You can ask whether you’d remain on your current medication during the trial while receiving treatment from the study team.

In double-blind trials, neither the participants nor the researchers know who is taking a placebo. These control groups help determine if the psychological effect of unknowingly taking an inert (inactive) treatment is as effective as the investigational drug itself. Double-blinding helps to avoid bias and ensure that measures regarding outcomes are as accurate as possible.¹

5. Like All Drugs, Investigational Drugs Have Potential Risks

Every drug brings a risk of possible side effects, and clinical trials are no exception. Before joining a clinical trial, you’ll be advised of possible side effects, or adverse reactions.⁸

It’s also important to be aware that if you’re given the investigational drug, it may not be effective.⁸

6. You Can Leave a Clinical Trial at Any Time

Enrollment in a clinical trial is 100 percent voluntary, and participants have the right to withdraw at any point.⁶

See If You May Be Eligible For A Clinical Trial

Talk With Others Who Understand

MyEpilepsyTeam is the social network for people with epilepsy and their loved ones. On MyEpilepsyTeam, thousands of members come together to ask questions, give advice, and share their stories with others who understand life with epilepsy.

Do you or your loved one have epilepsy and interest in joining a clinical trial? Have you participated in a clinical trial before? Share your experience in the comments below, or start a conversation by posting on your Activities page.