Study on Clemizole Hydrochloride for Children and Adults with Dravet Syndrome

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What is this study about?

This clinical trial is focused on studying Dravet Syndrome, a rare and severe form of epilepsy that begins in infancy. The trial will test a treatment called EPX-100, which is the code name for a medication known as clemizole hydrochloride. This medication is being tested as an additional therapy to help manage seizures in both children and adults who have Dravet Syndrome. Participants in the study will receive either the EPX-100 treatment or a placebo, which looks like the treatment but does not contain the active medication.

The purpose of the study is to evaluate how effective EPX-100 is in reducing the frequency of seizures in participants with Dravet Syndrome. The study will last for 20 weeks and will be conducted in a way that neither the participants nor the researchers know who is receiving the actual medication or the placebo, a method known as “double-blind.” This helps ensure that the results are not influenced by expectations about the treatment. Participants will take the medication orally, meaning it is taken by mouth in the form of a solution.

Throughout the study, participants will be monitored to see how their seizure frequency changes compared to before they started the treatment. The trial aims to provide valuable information on whether EPX-100 can be a helpful addition to the current treatment options for managing seizures in individuals with Dravet Syndrome.

1 joining the trial

Upon joining the trial, you will be required to provide written informed consent. This means you agree to participate after being informed about the nature and risks of the study.

You will need to meet certain criteria, such as having a clinical diagnosis of Dravet Syndrome and being on a stable regimen of anti-epileptic drugs (AEDs) for at least 30 days before the first visit.

2 observational phase

During this phase, you will maintain a daily seizure calendar to record any seizures you experience. This helps establish a baseline for your condition.

This phase lasts for 4 weeks, and it is important to keep an accurate record of your seizures during this time.

3 titration period

In this phase, you will start taking the study medication, EPX-100 (clemizole hydrochloride), or a placebo. The medication is administered as an oral solution.

The dosage will be gradually increased to determine the most effective and safe dose for you. This period is crucial for adjusting to the medication.

4 maintenance period

Once the optimal dose is reached, you will continue taking the medication at this dose. This phase is designed to maintain the effects of the medication.

You will continue to record any seizures in your daily calendar to monitor changes in seizure frequency.

5 end of trial

At the end of the 20-week trial, your participation will conclude. You may be asked to return for a final assessment to evaluate the effects of the medication.

The results of your participation will contribute to understanding the efficacy of EPX-100 in treating Dravet Syndrome.

Who Can Join the Study?

Participants must be 2 years or older.
Must have a clinical diagnosis of Dravet Syndrome.
Seizures must have started before 18 months of age.
Must have had normal development when seizures began.
Must have a history of specific types of seizures: generalized, unilateral clonic, and/or hemiclonic.
Must have a brain MRI showing no cortical malformation (no abnormal brain structure).
Must have a documented genetic mutation of the SCN1A gene.
Must be approved by the Principal Investigator (PI) after reviewing medical history and seizure diaries.
Must have a certain number of countable convulsive seizures during a 4-week baseline period.
Must be on a stable regimen of anti-epileptic drugs (AEDs) for at least 30 days before the first visit.
Must be in generally good health.
A parent or Legally Authorized Representative (LAR) must be able and willing to keep a daily seizure calendar.
Sexually active women who can become pregnant must use a medically acceptable method of birth control and have a negative pregnancy test at the screening visit.
A parent or LAR must be available and willing to give written informed consent after understanding the study’s nature and risks.

Who Cannot Join the Study?

Participants cannot join if they do not have Dravet syndrome.
Participants must be between the ages of 2 and 18 years old.
Participants cannot join if they are not experiencing countable convulsive seizures.
Participants cannot join if they are unable to follow the study procedures.
Participants cannot join if they have any other medical condition that might interfere with the study.
Participants cannot join if they are pregnant or breastfeeding.
Participants cannot join if they are taking certain medications that might interfere with the study.
Participants cannot join if they have a history of drug or alcohol abuse.
Participants cannot join if they have participated in another clinical trial recently.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country
Centrum Medyczne Plejady Magdalena Celinska Loewenhoff Michal Zolnowski sp.k.	Cracow	Poland
Instytut Matki I Dziecka	Warsaw	Poland
University Of Debrecen	Debrecen	Hungary
Semmelweis University	Budapest	Hungary
Mcfvbwp Utqshfbieu Ob Gozzms	Gdansk	Poland
Hesrcijt Dx Ly Shuaj Ciob I Sixr Pcm	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Hungary	Recruiting	01.03.2023
Poland	Recruiting	01.03.2023
Spain	Recruiting	01.03.2023

Trial locations

Investigated drugs:

Clemizole Hydrochloride

EPX-100 is a medication being tested in this clinical trial to see if it can help reduce seizures in people with Dravet Syndrome. Dravet Syndrome is a rare form of epilepsy that starts in childhood and can cause frequent and severe seizures. EPX-100 is being used as an additional treatment, meaning it is given alongside other medications that the participants are already taking for their condition. The goal of the trial is to find out if EPX-100 can lower the number of convulsive seizures that participants experience compared to when they started the trial.

Dravet syndrome – Dravet syndrome is a rare genetic disorder that begins in infancy and is characterized by frequent, prolonged seizures. These seizures often start as febrile seizures, which are triggered by fever or illness. As the child grows, the seizures may become more varied, including myoclonic, tonic-clonic, and absence seizures. The condition is associated with developmental delays and cognitive impairment, which can become more apparent over time. Children with Dravet syndrome may also experience behavioral challenges and difficulties with coordination and balance. The progression of the disease can vary, but it typically involves ongoing seizure activity and developmental challenges.

Trial ID:

2024-518628-57-00

Protocol code:

EPX-100-001

NCT ID:

NCT00945880

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Clemizole Hydrochloride for Children and Adults with Dravet Syndrome

What is this study about?

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