Epilepsy Talk

Clinical Trials Explained | May 7, 2016

New therapies, including medications, medical devices and surgical procedures are evaluated in research studies known as clinical trials.

Participation in clinical trials offers the potential for new treatment options and allows patients to participate with researchers in driving the discovery of effective therapies for epilepsy.

However, there are always risks involved, some known and possibly some that are unknowns, and patients should consult with their physicians in considering the appropriateness of a clinical trial for their particular situation.

Clinical trials are done under the supervision of the FDA.

Clinicaltrials.gov Provides regularly updated information about federally and privately supported clinical research in human volunteers.

About Clinical Research Shows how experimental drugs are tested, who pays for the research and how to participate in clinical trials.

Newly Approved Drugs Gives information about which medications have recently been approved by the FDA.

Research Center Profiles Gives profiles of research centers, organized by geographic region.

Clinical Trial Results Gives you easy access to information about the results of recently completed and ongoing clinical trials.

Learn how experimental drugs are tested, who pays for the research and how to participate in clinical trials.

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 others 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.

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?

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 follow 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 the treatment plan will entail.

The type and frequency of tests.

How the results will be measured.

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.

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.

Frequently Asked Questions About Epilepsy Research

http://www.epilepsyhero.org/faq/

Courtesy of The Epilepsy Foundation of America

http://www.epilepsy.com/learn/treating-seizures-and-epilepsy/clinical-trials

 

To subscribe to EpilepsyTalk.com. and received the latest articles by email, simply enter your name in the bottom right hand box and click on “Follow”

 

 

 

---