RecruitingPHASE3INTERVENTIONAL

A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome

This research is testing a new medicine, EPX-100 (also known as clemizole hydrochloride), for people with Dravet syndrome. Dravet syndrome is a type of epilepsy that causes severe seizures. The study wants to find out if EPX-100 can reduce seizures and if it's safe for children and adults who are already taking other seizure medications. Some participants will receive the active medicine, while others will receive a placebo (dummy medicine), so we can compare the effects. Researchers are carefully monitoring participants to understand how well the new medicine works and if there are any side effects. This is a "Phase 3" study, which means it's one of the final steps before a medicine might be approved for wider use.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Epygenix

Enrolment target

150

Start

15 Sep 2020

Estimated completion

01 May 2029

Dravet Syndrome

What is this study about?

Dravet syndrome is a challenging type of epilepsy that often begins in infancy and continues throughout life. It causes frequent and prolonged seizures, which can be difficult to control, even with many medications. Because of this, doctors and researchers are always looking for new and better ways to help people living with Dravet syndrome.

This study is focusing on a medicine called EPX-100, or clemizole hydrochloride. The main goal is to see if adding EPX-100 to a person's current seizure medications (this is called "adjunctive therapy") can help reduce the number of seizures they experience. We also want to make sure it's safe to use. By comparing EPX-100 to a placebo, which looks exactly like the real medicine but contains no active drug, researchers can get a clear picture of how effective EPX-100 truly is.

This is a large, worldwide study taking place in many different centres. It's a "Phase 3" study, meaning it's one of the final stages of testing before a new medicine might be considered for approval. This phase involves a larger group of people to confirm the findings from earlier studies and gather more information about safety and effectiveness over a longer period.

Key takeaways

Tests a new medicine (EPX-100) for Dravet syndrome alongside current treatments.
Aims to see if EPX-100 can reduce seizures and is safe.
Involves children and adults aged 2 years and older.
Compares EPX-100 to a dummy medicine (placebo) in a fair way.
Participation includes observation, a 16-week main study, and an optional longer-term period.
You can stop participating at any time if you change your mind.

Who may be eligible?

To join this study, participants must be at least 2 years old. A parent or legal guardian must agree for children to take part, and adults must agree themselves. Everyone involved needs to have a clear diagnosis of Dravet syndrome and still be experiencing seizures, even if they're taking other medicines.

There are also some specific medical requirements for those with Dravet syndrome. These include having had seizures before 18 months of age, showing normal development at the beginning, and a specific genetic change (mutation) in something called the SCN1A gene. Your doctor will be able to check all these details.

However, some things would mean you can't join the study. If you've had an allergic reaction to clemizole HCl before, or if you're taking part in another drug study, you wouldn't be able to participate. Also, certain other health conditions, specific medications (like lorcaserin or fenfluramine), or recent brain surgery for epilepsy would prevent you from joining.

Quick self-check

Are you, or the person you care for, 2 years old or older?
Do you have a clear diagnosis of Dravet syndrome with ongoing seizures?
Did seizures start before 18 months of age with normal development at that time?
Has a specific genetic change (SCN1A gene mutation) been identified?
Are you willing to attend regular study appointments?
Have you *not* had a known allergic reaction to clemizole HCl in the past?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, the study will involve several stages. First, there will be an "Observational Period" lasting about 4 weeks. During this time, the study team will get to know your usual seizure patterns without making any changes to your medication.

After that, you'll enter the "Double-Blind Period" for 16 weeks. In this part, you'll be given either the study medicine (EPX-100) or a placebo, but neither you, your doctor, nor the study team will know which you are receiving. This helps make the study fair and accurate. You'll continue to take your regular seizure medications as well.

After the 16 weeks, there's an "Open-Label Extension (OLE) Period." In this stage, everyone who completes the initial period will have the chance to receive the actual study medicine (EPX-100) if they wish, and everyone will know what they are taking. Throughout all periods, you'll have regular visits with the study team for check-ups, to monitor your seizures, and to make sure you're feeling well. The total length of your participation will depend on how long you stay in the open-label extension.

Potential risks and benefits

Taking part in any medical study has both potential benefits and risks. You might benefit if EPX-100 helps reduce your seizures. You would also be contributing important information that could help others with Dravet syndrome in the future. However, there's a chance you might experience side effects from the study medication, or you might receive the placebo and therefore not see any improvement in your condition. You are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (58)

University of Alabama at Birmingham
Birmingham, United States· Active not recruiting
Arkansas Children's Hospital
Little Rock, United States· Active not recruiting
Children's Hospital of Los Angeles
Los Angeles, United States· Terminated
University of California Irvine
Orange, United States· Recruiting
UCSF Medical Center
San Francisco, United States· Recruiting
Yale University School of Medicine
New Haven, United States· Not yet recruiting
The Nemours Foundation
Wilmington, United States· Terminated
Rare Disease Research FL
Kissimmee, United States· Recruiting
Pediatric Neurology and Epilepsy Specialists
Winter Park, United States· Recruiting
Clinical Integrative Research Center of Atlanta (CIRCA)
Atlanta, United States· Withdrawn
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States· Terminated
Norton Children's Research Institute
Louisville, United States· Recruiting

+46 more sites — see the official record for the full list.

Common questions

What is Dravet syndrome?

Dravet syndrome is a severe type of epilepsy that typically starts in babies and causes frequent, long-lasting seizures that can be hard to control.

What does 'placebo-controlled' mean?

It means some people will get the actual medicine, and others will get a 'dummy' pill with no medicine in it. This helps researchers fairly compare the new medicine's effects.

What is a 'Phase 3' study?

Phase 3 is typically the final stage of testing a new medicine before it can be considered for approval. It involves more people to confirm its safety and effectiveness.

Can I continue my other seizure medicines?

Yes, this study is looking at EPX-100 as an 'add-on' treatment, meaning you will continue taking your existing seizure medications.

Will I know if I'm getting the real medicine or the placebo?

During the main part of the study, neither you nor your doctor will know. This is called 'double-blind.' Later, in the 'open-label' part, everyone knows they are taking the active medicine.

How to find out more

Krystle Rapchak

clinicaltrials@harmonybiosciences.com +1 (312) 847-1289 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.