RecruitingRare disease

Study on Clemizole Hydrochloride for Children and Adults with Dravet Syndrome

Fast replyInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 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.
Step 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.
Step 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.
Step 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.
Step 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 trial?

14 criteria

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 trial?

9 criteria

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.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Centrum Medyczne Plejady Magdalena Celinska Loewenhoff Michal Zolnowski sp.k.
Cracow, Poland
Verified
Hospital De La Santa Creu I Sant Pau
Barcelona, Spain
Responsive
Semmelweis University
Budapest, Hungary

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Investigated drugs

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.

Clemizole Hydrochloride

What is already known about the treatment

EPX-100 (Clemizole Hydrochloride) – EPX-100 is administered orally and is being studied as an additional treatment for Dravet syndrome, a severe form of epilepsy. Currently, it is in the clinical trial phase, specifically in a 20-week study to assess its effectiveness in reducing seizure frequency. The medication works by targeting specific receptors in the brain to help stabilize electrical activity, which may reduce the number of seizures. It is classified as an investigational antiepileptic drug, meaning it is still being researched and is not yet widely available for general medical use.

Investigated diseases

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.