In short

There are currently 5 ongoing clinical trials investigating new treatments for epileptic encephalopathy. These studies are evaluating different investigational medications for various forms of developmental and epileptic encephalopathies, including Dravet Syndrome, Lennox-Gastaut Syndrome, and genetic forms related to SCN2A, SCN8A, and KCNT1 mutations. The trials are being conducted across several European countries, with participants ranging from young children to adults.

Clinical trial locations

Study on the Safety and Effectiveness of Bexicaserin for Seizures in Children and Adults with Developmental and Epileptic Encephalopathy

This trial is investigating bexicaserin, also known as LP352, as a treatment for people with Developmental and Epileptic Encephalopathies. These are severe brain disorders that typically start in childhood and cause frequent seizures along with developmental delays.

Main inclusion criteria: Participants must have completed a previous study of bexicaserin and be considered likely to benefit from continued treatment. They must have a confirmed diagnosis of developmental and epileptic encephalopathy, including conditions such as Dravet Syndrome, Lennox-Gastaut Syndrome, or other related forms. Participants need a reliable caregiver or guardian who can assist throughout the study and maintain a seizure diary. Both children and adults of any gender can participate.

Main exclusion criteria: People without a diagnosis of developmental and epileptic encephalopathy cannot participate. Those who cannot follow study procedures, have interfering medical conditions or medications, have a history of allergic reactions to similar medications, or are pregnant or breastfeeding are excluded. Recent participation in other clinical trials also prevents enrollment.

Trial focus: The study evaluates the long-term safety and effectiveness of bexicaserin in reducing seizure frequency. Participants receive the medication as an oral solution over an extended period, with regular monitoring through physical examinations, vital sign checks, and laboratory tests. Participants or their caregivers keep detailed seizure diaries to track treatment response.

Investigational drug: Bexicaserin (LP352) is a serotonin receptor modulator given as an oral solution. It works by modulating specific brain receptors to help stabilize neuronal activity and reduce seizure frequency.

Title: Study of LP352 (bexicaserin) oral solution versus placebo for treating seizures in children and adults with Developmental and Epileptic Encephalopathies

This study focuses on evaluating whether bexicaserin can effectively reduce seizures in people with Developmental and Epileptic Encephalopathies, particularly Lennox-Gastaut Syndrome.

Main inclusion criteria: Participants must have Lennox-Gastaut Syndrome with seizures starting before age 8, history of tonic seizures and other seizure types, and developmental delays, or other forms of developmental and epileptic encephalopathy with seizures starting before age 5. They must experience at least 4 countable motor seizures per month and be on stable doses of 1-4 anti-seizure medications. Participants must be able to maintain a seizure diary throughout the study.

Main exclusion criteria: People younger than 2 or older than 65, those with recent status epilepticus (prolonged seizures), those taking more than 4 anti-seizure medications, and those with severe liver or kidney disease cannot participate. Pregnant or breastfeeding women, those with unstable medical conditions, significant heart problems, or severe psychiatric disorders are also excluded.

Trial focus: This is a placebo-controlled study lasting approximately 17 weeks. Some participants receive bexicaserin while others receive placebo. The study measures changes in seizure frequency, particularly motor seizures including tonic-clonic, tonic, atonic, and focal seizures. Participants continue their regular anti-seizure medications during the trial.

Investigational drug: LP352 (bexicaserin) is given as an oral solution with a maximum daily dose of 36 milligrams. It is being tested to determine if it can reduce the frequency of motor seizures in people with developmental and epileptic encephalopathies.

Study of Elsunersen in Children with SCN2A Developmental and Epileptic Encephalopathy to Reduce Seizures

This trial is studying elsunersen, also known as PRAX-222, for children with a specific genetic form of epileptic encephalopathy caused by mutations in the SCN2A gene.

Main inclusion criteria: Children must have confirmed SCN2A gene mutations through genetic testing and first seizures before 3 months of age. Participants must be between newborn (at least 37 weeks gestation) and 18 years old, with at least 4 countable motor seizures within 28 days. They need normal kidney function and liver function tests within acceptable ranges. Brain MRI results and seizure frequency must be reviewed by an Eligibility Review Committee. Participants must complete seizure diary entries for at least 80% of days during screening.

Main exclusion criteria: Children younger than 2 or older than 17, those without confirmed SCN2A mutation, those with severe allergic reactions to similar medications, and those with severe liver or kidney problems cannot participate. Pregnant or breastfeeding individuals, those who participated in other trials within 30 days, and those who had major surgery in the past 3 months are excluded.

Trial focus: The study uses a double-blind design where some children receive elsunersen while others undergo a sham procedure. The treatment period lasts 24 weeks, followed by an extension phase. The medication is given through intrathecal injection (into the spinal fluid). Researchers monitor seizure frequency, developmental progress, and safety throughout the study.

Investigational drug: Elsunersen is designed to target the SCN2A gene mutations that cause seizures. It is administered through injections into the spinal fluid at specific intervals, with a maximum dose of 1 mg per administration and 12 mg total over 48 weeks.

Study on PRAX-562 for Children with Developmental and Epileptic Encephalopathies

This trial is investigating PRAX-562 for children with specific genetic forms of epileptic encephalopathy related to SCN2A and SCN8A gene mutations.

Main inclusion criteria: Children between 2 and 18 years old weighing at least 10 kg can participate. They must have documented genetic variants in SCN2A with seizures starting in the first 3 months of life, or SCN8A-related developmental and epileptic encephalopathy confirmed by genetic testing in an accredited laboratory. Participants must have at least 8 countable motor seizures in the 4 weeks before screening and during the 28-day baseline observation period. They must be on stable doses of anti-seizure medications for at least 1 month before screening and be able to maintain seizure diaries.

Main exclusion criteria: Children without confirmed SCN2A or SCN8A diagnosis, those outside the 2-18 age range, those unable to follow study procedures, and those with significant changes in seizure medications before the study cannot participate. Those with drug or alcohol abuse history, pregnant or breastfeeding individuals, those with allergies to study medication, and those currently in other trials are excluded.

Trial focus: The study has two parts. First, participants are randomly assigned to receive either PRAX-562 or placebo in a double-blind phase. The second part is an open-label extension where all participants receive PRAX-562. This allows researchers to gather information about long-term safety and effects. Throughout the study, researchers monitor seizure frequency and any side effects.

Investigational drug: PRAX-562 is a powder for oral suspension that can be taken by mouth or through a feeding tube. It targets specific sodium channels in the brain involved in transmitting electrical signals, aiming to stabilize these signals and reduce seizure activity.

Summary

These 5 clinical trials represent important research efforts to develop new treatments for various forms of epileptic encephalopathy. Two trials are investigating bexicaserin (LP352) across multiple European countries including Germany, Spain, Latvia, France, Belgium, Italy, Portugal, and the Netherlands. These studies focus on both broad categories of developmental and epileptic encephalopathies and specifically on Lennox-Gastaut Syndrome.

Three trials are targeting specific genetic forms of the condition. Elsunersen is being studied in Germany and Italy for SCN2A-related cases, while S230815 is being tested in Spain and France for KCNT1-related cases. PRAX-562 is being investigated in Spain for both SCN2A and SCN8A-related forms.

The trials use different approaches to medication delivery. Bexicaserin and PRAX-562 are given orally, while elsunersen and S230815 require intrathecal injection directly into the spinal fluid. Most trials include children, with some also accepting adults, reflecting the typical age range when these conditions manifest and require treatment.

All studies require participants to maintain seizure diaries and continue their existing anti-seizure medications while receiving the investigational treatments. This approach allows researchers to evaluate how well the new medications work as add-on therapies to standard treatment.