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Severe myoclonic epilepsy of infancy – Trials in Disease

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Ongoing Clinical Trials for Severe Myoclonic Epilepsy of Infancy

There are currently 9 clinical trials underway testing new treatments for severe myoclonic epilepsy of infancy (also known as Dravet Syndrome). These studies are being conducted across multiple European countries and are evaluating several investigational medications aimed at reducing seizure frequency and improving quality of life for patients with this rare and severe form of epilepsy.

Clinical trial locations

Study on Clemizole Hydrochloride for Children and Adults with Dravet Syndrome

This trial is testing clemizole hydrochloride, known as EPX-100, as an additional treatment for seizures in people aged 2 years and older. The study compares the medication to a placebo over a 20-week period to see if it can reduce the frequency of convulsive seizures.

Main inclusion criteria: Participants must be at least 2 years old with a clinical diagnosis confirmed by a documented SCN1A gene mutation. Seizures must have started before 18 months of age in a child who was developing normally at that time. Participants need to have experienced specific types of seizures including generalized, unilateral clonic, or hemiclonic seizures. They must be taking stable doses of anti-epileptic drugs for at least 30 days before joining and have a certain minimum number of countable convulsive seizures during the baseline period. A brain MRI showing no cortical malformation is required.

Main exclusion criteria: Those without a confirmed diagnosis cannot participate. Participants who are pregnant, breastfeeding, or unable to follow study procedures are excluded. Those taking certain medications that might interfere with the study or who have a history of drug or alcohol abuse are also not eligible. Recent participation in another clinical trial is an exclusion factor.

Trial focus: The study aims to evaluate whether EPX-100 can effectively lower the number of convulsive seizures compared to placebo. It uses a double-blind design where neither participants nor researchers know who receives the actual medication.

Investigational drug: EPX-100 is an oral solution taken by mouth, being tested to see if it can reduce seizure frequency when added to existing treatments.

Study on the Effects of Bexicaserin for Treating Seizures in Children and Adults with Dravet Syndrome

This trial evaluates bexicaserin, also called LP352, as an oral solution for reducing countable motor seizures. The study compares it to placebo in participants aged 2 to 65 years.

Main inclusion criteria: Participants must be between 2 and 65 years old with a confirmed diagnosis. Seizures must have started between 1 and 20 months of age in an otherwise healthy infant. A history of specific seizure types is required, including prolonged generalized tonic-clonic, hemiclonic, myoclonic, tonic, atonic, atypical absence, focal awareness, or nonconvulsive status epilepticus. Participants must have experienced at least 4 countable motor seizures per month for the 3 months before enrollment and be taking 1 to 4 anti-seizure medications at stable doses for at least 4 weeks.

Main exclusion criteria: Those with medical conditions other than the target diagnosis, those outside the specified age range, pregnant or breastfeeding individuals, and those with recent participation in another trial are excluded. A history of drug or alcohol abuse also prevents participation.

Trial focus: The study monitors seizure frequency and maintains daily seizure diaries to determine if LP352 can reduce motor seizures effectively and safely.

Investigational drug: LP352 is an oral solution being tested for its ability to reduce motor seizures while being evaluated for safety and tolerability.

Study of zorevunersen to reduce seizures in patients with Dravet syndrome

This trial tests zorevunersen, also known as STK-001, which is given as an injection into the spinal fluid. The study aims to reduce major motor seizures over a 52-week treatment period.

Main inclusion criteria: Participants must be between 2 and 18 years old with a clinical diagnosis confirmed by a specific genetic variant in the SCN1A gene. Seizures must have started before 12 months of age with normal development at onset and no other known cause for symptoms. A required number of major motor seizures during a 6-week observation period is necessary. Participants must have tried at least 2 previous seizure treatments and be currently taking at least one anti-seizure medication. All current treatments including anti-seizure medications, ketogenic diet, vagus nerve stimulation, or cannabinoid products must remain stable.

Main exclusion criteria: Those below 2 years old, without confirmed genetic diagnosis, not experiencing major motor seizures, or with severe liver or kidney problems cannot participate. Pregnancy, breastfeeding, history of severe allergic reactions, current participation in other trials, or inability to follow study procedures are also exclusion factors.

Trial focus: The study measures major motor seizure frequency changes between weeks 16 and 28, comparing zorevunersen to a sham treatment while participants continue their regular medications.

Investigational drug: Zorevunersen is administered through intrathecal injection and works by targeting SCN1A gene expression to help restore normal sodium channel function in brain cells.

Study on the Safety and Effects of Fenfluramine in Infants Aged 1 to Less Than 2 Years with Dravet Syndrome

This trial evaluates the safety and tolerability of fenfluramine hydrochloride, known as ZX008, in very young children aged 1 to less than 2 years. The oral solution is monitored over up to 52 weeks.

Main inclusion criteria: Children must be at least 1 year old but less than 2 years old with a documented or likely diagnosis. They must be taking at least one antiseizure medication at a stable dose for at least 4 weeks before screening. At least one countable motor seizure during the baseline period is required. Participants must weigh at least 8 kilograms.

Main exclusion criteria: Infants without the diagnosis, those outside the age range, with other interfering medical conditions, or taking medications that might interfere cannot participate. Serious allergic reactions to similar medications and other health issues deemed unsafe by study doctors are exclusion factors.

Trial focus: The study focuses on understanding how the body processes the medication and ensuring it is safe for young children, monitoring heart function, growth patterns, and seizure frequency.

Investigational drug: Fenfluramine is an oral solution with dosage ranging from 0.2 to 0.8 mg per kilogram of body weight per day, administered alongside existing antiseizure medications.

Long-Term Safety Study of Fenfluramine Hydrochloride for Seizures in Patients with Dravet or Lennox-Gastaut Syndrome

This trial assesses the long-term safety of fenfluramine hydrochloride, also known as ZX008, as an additional therapy for managing seizures. It is designed for participants who have completed a previous core study.

Main inclusion criteria: Participants must be male or nonpregnant, nonlactating females who have satisfactorily completed a core study and successfully completed another clinical trial with ZX008. Caregivers must be willing and able to follow study procedures, attend scheduled visits, and manage the study medication properly.

Main exclusion criteria: Those without specific diagnoses, outside the specified age range, not part of the specified clinical trial groups, or with conditions that make participation unsafe are excluded.

Trial focus: The study evaluates long-term safety and tolerability through regular monitoring including laboratory tests, vital signs, electrocardiograms, and specialized heart function tests. Effectiveness is assessed through caregiver and investigator evaluations.

Investigational drug: ZX008 is an oral solution used as adjunctive therapy, influencing serotonin levels in the brain to help reduce seizure frequency and severity.

Study on Cannabidiol Oral Solution for Infants with Tuberous Sclerosis, Dravet Syndrome, or Lennox-Gastaut Syndrome Experiencing Uncontrolled Seizures

This open-label trial studies cannabidiol oral solution, known as GWP42003-P or Epidyolex, in young children with epilepsy whose seizures are difficult to control. The 52-week study monitors safety and effectiveness.

Main inclusion criteria: Children with a specific diagnosis between 1 year and less than 2 years old can participate. Parents or legal representatives must be willing to notify authorities about participation if required, inform the child’s primary care doctor, and complete electronic diaries. Seizures must not be adequately controlled with current medications. A suitable video electroencephalogram must be available or completed, and participants must be taking at least one anti-seizure medication at stable doses for 2 weeks before treatment begins.

Main exclusion criteria: History of severe allergic reactions to the medication, pregnancy, breastfeeding, significant liver or kidney disease, recent participation in another trial, substance abuse history, other unsafe medical conditions, or inability to comply with study procedures prevent participation.

Trial focus: The study evaluates safety, effectiveness, and how the body processes cannabidiol. Caregivers maintain seizure diaries every four weeks, and blood samples are taken to measure medication levels.

Investigational drug: Cannabidiol oral solution is taken by mouth as an additional treatment, interacting with the endocannabinoid system in the brain to potentially reduce seizure frequency.

Study on Long-Term Safety of Soticlestat for Adults and Children with Developmental Epileptic Encephalopathies

This extension study evaluates the long-term safety of soticlestat, also known as TAK-935, in participants who have already been involved in earlier research. The treatment period can last up to 96 weeks with a maximum daily dose of 600 milligrams.

Main inclusion criteria: Participants must have been part of a previous soticlestat study and either successfully completed it or received at least 10 weeks of treatment. They must not have experienced serious or severe side effects that would make continued use unsafe. The study doctor must believe the participant could benefit from taking soticlestat.

Main exclusion criteria: Those with medical conditions other than the specific types of epileptic encephalopathies being studied or not using at least one anti-seizure therapy cannot participate. Those outside the specified age range, unwilling or unable to follow study procedures, or with other conditions making participation unsafe are excluded.

Trial focus: The study monitors adverse events, changes in behavior, vital signs, body weight, and laboratory tests. Behavioral and functional measures are evaluated using specialized scales.

Investigational drug: Soticlestat is administered orally as a tablet alongside other anti-seizure therapies, targeting specific enzymes in the brain to help reduce seizure frequency.

Study on Long-Term Safety of Soticlestat for Patients with Dravet or Lennox-Gastaut Syndromes

This extension study assesses the long-term safety and tolerability of soticlestat over up to 52 weeks for participants who completed a previous phase 3 study and received at least 12 weeks of treatment.

Main inclusion criteria: Participants must have been part of a previous phase 3 soticlestat study with at least 12 weeks of treatment and no serious adverse effects. The doctor must believe the participant could benefit from continued treatment. Participants or their caregivers must be able to complete assessments, keep daily seizure records, and take the medication as directed. Female participants who can have children must have a negative pregnancy test and agree to use effective birth control.

Main exclusion criteria: Those without specific diagnoses, outside the specified age range, unable to follow the treatment plan including additional therapies, or unable to safely take soticlestat cannot participate.

Trial focus: The study monitors health through regular assessments including vital signs, laboratory tests, and heart evaluations. Participants or caregivers maintain daily seizure diaries to track changes in seizure frequency.

Investigational drug: Soticlestat is taken orally as a tablet alongside standard treatments including anti-seizure medications, vagus nerve stimulation, ketogenic diet, or modified Atkins diet.

Study on Soticlestat for Children and Adults with Dravet or Lennox-Gastaut Syndrome Previously Treated with Fenfluramine

This trial evaluates soticlestat in participants aged 2 years or older who have previously been exposed to fenfluramine. The study lasts up to 52 weeks and compares the medication to placebo.

Main inclusion criteria: Participants must be at least 2 years old with a documented clinical diagnosis and previous exposure to fenfluramine, either currently taking it or having used it in the past. A baseline seizure frequency is established over a 28-day period.

Main exclusion criteria: Those without the specific diagnoses, not previously exposed to fenfluramine, outside the age range, or not part of the specified trial group cannot participate.

Trial focus: The primary endpoint measures the percent change in seizure frequency over a 12-week maintenance period to determine the effectiveness of soticlestat in reducing seizures.

Investigational drug: Soticlestat is administered orally as a tablet, targeting specific brain pathways to help reduce seizure frequency and severity.

Summary

The 9 ongoing clinical trials for severe myoclonic epilepsy of infancy are concentrated in several European countries, with Spain hosting the most trials, followed by Italy and Poland. The trials are testing diverse treatment approaches including clemizole hydrochloride, bexicaserin, zorevunersen, fenfluramine, cannabidiol, and soticlestat. Several studies focus on long-term safety evaluation of medications in extension trials for participants who have already completed earlier research phases. Most trials require participants to maintain stable doses of their existing anti-seizure medications while adding the investigational treatment. Age ranges vary considerably, with some trials focused specifically on very young children aged 1 to less than 2 years, while others include participants from early childhood through adulthood. The investigational drugs work through different mechanisms, including serotonin modulation, gene expression targeting, and enzyme inhibition, reflecting the variety of scientific approaches being explored to manage this challenging condition.

Ongoing Clinical Trials on Severe myoclonic epilepsy of infancy

  • Study on the Effects of Bexicaserin for Treating Seizures in Children and Adults with Dravet Syndrome

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    Belgium Czechia Denmark Finland France Germany +5

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