Not recruitingRare disease

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

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 a group of rare conditions known as developmental and epileptic encephalopathies (DEEs), specifically those related to genetic changes in the SCN2A and SCN8A genes. These conditions are characterized by severe epilepsy and developmental delays in children. The trial is testing a new treatment called PRAX-562, which is a powder that can be taken by mouth. The purpose of the study is to explore the safety and how well children tolerate this new treatment, as well as its effects on reducing the frequency of seizures.

The study is divided into two parts. In the first part, participants will be randomly assigned to receive either PRAX-562 or a placebo, which looks like the treatment but does not contain the active substance. This part of the study is double-blind, meaning neither the participants nor the researchers know who is receiving the actual treatment. The second part of the study is an open-label extension, where all participants will receive PRAX-562. This allows researchers to gather more information about the long-term safety and effects of the treatment.

Participants in the study will be children aged 2 to 18 years who have been diagnosed with SCN2A or SCN8A related DEEs. The study will monitor the number of seizures the children experience and any side effects they may have while taking the treatment. The trial aims to provide valuable insights into the potential benefits of PRAX-562 for children with these challenging conditions.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the clinical trial, an initial assessment is conducted to confirm eligibility. This involves reviewing medical history and current health status.
Participants must have a documented variant in SCN2A or a diagnosis of SCN8A-DEE, with seizures starting in the first three months of life.
Step 2
Baseline observation period
A 28-day baseline observation period is required. During this time, participants or their guardians must record seizure frequency in a daily seizure diary.
This period helps establish a baseline for comparison during the trial.
Step 3
Randomized treatment phase
Participants are randomly assigned to receive either the investigational medication PRAX-562 or a placebo. This phase is double-blind, meaning neither participants nor researchers know who receives the actual medication.
The medication is administered as a powder for oral suspension, which can be taken orally or through a nasogastric or percutaneous endoscopic gastrostomy tube.
Step 4
Treatment administration
The medication is taken according to the prescribed dosage and frequency, as determined by the study protocol. Participants must adhere to the schedule for the duration of the trial.
Regular monitoring and assessments are conducted to evaluate safety, tolerability, and efficacy.
Step 5
Open-label extension
After completing the randomized phase, participants may enter an open-label extension where all receive PRAX-562.
This phase allows for the evaluation of long-term safety and tolerability.
Step 6
Ongoing monitoring
Throughout the trial, participants undergo regular health assessments and monitoring to track any changes in seizure frequency and overall health.
Participants are required to maintain stable doses of any other antiseizure medications and therapies unless instructed otherwise by the study investigator.
Step 7
Completion and follow-up
Upon completion of the trial, a final assessment is conducted to evaluate the overall impact of the treatment.
Participants may be asked to continue providing health updates for a specified period to monitor any long-term effects.

Who can join the trial?

11 criteria

The participant, or their parent/legal guardian, must be willing to sign a consent form, showing they understand the purpose of the study and agree to follow all procedures, including keeping a seizure diary.
The participant must be willing and able to keep all seizure treatments stable during the study, unless advised otherwise by the study doctor.
If the participant is sexually active or can have children, they must agree to use a method of birth control as defined in the study.
The participant must have a documented genetic variant in SCN2A with seizures starting in the first 3 months of life, or a diagnosis of SCN8A-DEE supported by clinical and genetic findings. Genetic testing must be done in an accredited laboratory.
The participant must be between the ages of 2 and 18 years at the time of screening.
The participant must weigh at least 10 kg at the time of screening.
The participant must have had at least 8 countable motor seizures in the 4 weeks before screening and at least 8 countable motor seizures during the 28-day baseline observation period, as recorded in a seizure diary.
The participant must be assessed as suitable for the study by the study doctor and an Eligibility Review Committee.
The participant must be on stable doses of anti-seizure medications for at least 1 month before screening. For some groups, no more than 1 or 2 of these medications can be sodium channel-blocking types.
If the participant uses vagus nerve stimulation (VNS), it must have been in place for at least 3 months before screening with stable settings for at least 1 month before screening. VNS is not counted as a medication.
If the participant is on a ketogenic or other special diet for seizure management, the diet must have started at least 3 months before screening with stable parameters for at least 1 month before screening. Diets are not counted as medications.

Who cannot join the trial?

9 criteria

Patients who do not have a diagnosis of **SCN2A** or **SCN8A developmental and epileptic encephalopathy** cannot participate. This is a specific type of epilepsy that starts in childhood and affects brain development.
Patients who are not within the age range of 2 to 18 years old cannot participate.
Patients who are not able to follow the study procedures or take the study medication as required cannot participate.
Patients who have other serious medical conditions that might interfere with the study cannot participate.
Patients who are currently participating in another clinical trial cannot participate.
Patients who have had a significant change in their seizure medications within a certain period before the study starts cannot participate.
Patients who have a history of drug or alcohol abuse cannot participate.
Patients who are pregnant or breastfeeding cannot participate.
Patients who have a known allergy to the study medication or its ingredients cannot participate.

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Responsive
Hospital Ruber Internacional
Madrid, Spain

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Investigated drugs

PRAX-562 is a medication being studied for its potential to help children with developmental and epileptic encephalopathies, which are conditions that cause seizures and affect brain development. The trial aims to see if PRAX-562 is safe and well-tolerated by children, and whether it can reduce the number of motor seizures they experience. In the initial part of the study, the medication is given in a controlled, blinded manner to compare its effects with those of a placebo. In the later part, all participants receive PRAX-562 openly to assess its long-term safety and effectiveness.

What is already known about the treatment

PRAX-562 – PRAX-562 is administered orally and is currently being studied in a clinical trial to assess its safety, tolerability, and effectiveness in children with developmental and epileptic encephalopathies, specifically those related to SCN2A and SCN8A genes. This medication is in the experimental phase and is not yet widely recognized in medical literature as a standard treatment. It is primarily being investigated for its potential to reduce the frequency of seizures in affected children. PRAX-562 works by targeting specific channels in the brain that are involved in the transmission of electrical signals, aiming to stabilize these signals and reduce seizure activity. It is classified as an investigational antiepileptic drug.

Investigated diseases

SCN2A developmental and epileptic encephalopathy (SCN2A DEE)
This condition is a severe neurological disorder characterized by early-onset seizures and developmental delays. It is caused by mutations in the SCN2A gene, which affects the function of sodium channels in the brain. The disease typically begins in infancy or early childhood, with frequent and varied types of seizures. As the condition progresses, affected individuals may experience significant cognitive and motor impairments. The severity and specific symptoms can vary widely among individuals. Over time, the condition can lead to challenges in daily functioning and require ongoing care.
SCN8A developmental and epileptic encephalopathy (SCN8A DEE)
This disorder is a rare genetic condition that leads to severe epilepsy and developmental issues. It results from mutations in the SCN8A gene, impacting the sodium channels that are crucial for normal brain activity. The disease often manifests in infancy or early childhood with frequent seizures that can be difficult to control. As it progresses, individuals may experience developmental delays, intellectual disabilities, and motor skill challenges. The symptoms and severity can differ significantly from person to person. The condition requires careful management to address the complex needs of those affected.