Active not recruitingPHASE2, PHASE3INTERVENTIONAL

A Clinical Trial of PRAX-562 in Subjects With Developmental and Epileptic Encephalopathies (DEE)

This research study is looking into a new medicine called PRAX-562 for young people aged 1 to 18 years old who have specific types of severe epilepsy known as SCN2A encephalopathy or SCN8A encephalopathy. These conditions are rare and often lead to frequent seizures. The main goal of the study is to find out if PRAX-562 is safe for these children and teenagers, and to see how well their bodies tolerate the medicine. It also aims to understand if the medicine can help reduce the number of seizures they experience. Participants will be given either PRAX-562 or a dummy medicine (placebo) for a period, and their health and seizures will be closely monitored. This is a crucial step in developing new treatments for these challenging conditions.

At a glance

Status

Active not recruiting

Phase

PHASE2, PHASE3

Sponsor

Praxis Precision Medicines

Enrolment target

Start

02 Aug 2023

Estimated completion

01 Mar 2027

SCN2A Encephalopathy SCN8A Encephalopathy

What is this study about?

This research study is about a new medicine called PRAX-562. It's being tested in children and teenagers, aged 1 to 18, who have a specific type of severe epilepsy. These conditions are called SCN2A encephalopathy and SCN8A encephalopathy. These types of epilepsy are caused by changes in certain genes and can lead to very frequent seizures and affect development.

The main purpose of this study is to see if PRAX-562 is safe for these young people and how well their bodies handle it. It also aims to see if the medicine can help reduce the number of seizures they have. Finding new and effective treatments for these challenging conditions is very important, as current options don't always work for everyone.

Participants in the study will be split into groups; some will receive PRAX-562, and others will receive a placebo (a dummy medicine that looks the same but has no active ingredient). This allows the researchers to fairly compare the effects of the new medicine. Everything will be done carefully, with doctors and nurses closely monitoring each participant's health and seizure activity throughout the study.

Key takeaways

This study is testing a new medicine (PRAX-562) for rare, severe forms of epilepsy (SCN2A & SCN8A encephalopathy).
It's for children and teenagers aged 1 to 18.
The main goals are to check the medicine's safety and how well it's tolerated, and if it reduces seizures.
Participants will receive either the new medicine or a dummy medicine.
Taking part involves regular clinic visits, seizure diaries, and medication.
You can withdraw from the study at any time without affecting your child's care.

Who may be eligible?

To be considered for this study, a child or teenager must be between 1 and 18 years old. They must have a confirmed diagnosis of SCN2A encephalopathy with seizures starting in their first three months of life, or a diagnosis of SCN8A encephalopathy based on both their symptoms and genetic tests.

Also, to be part of the study, the young person needs to have had at least 8 noticeable seizures in the four weeks before being screened for the study. They also need to have at least 8 noticeable seizures during a specific 28-day period where their seizures are carefully recorded in a diary. The study team will check for other specific requirements to ensure it's the right fit.

Some reasons a child or teenager might not be able to join include having other significant genetic conditions that contribute to their epilepsy or developmental issues. They also wouldn't be able to join if they have a specific type of genetic change that means certain seizure medicines might make their seizures worse. Frequent severe seizures needing hospitalisation and breathing support in the last six months would also generally exclude participation. There are other criteria the study team will review.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is your child aged between 1 and 18 years?
Does your child have a confirmed diagnosis of SCN2A encephalopathy (with seizures starting before 3 months old) or SCN8A encephalopathy?
Has your child recently had at least 8 noticeable seizures within a 4-week period?
Has your child had at least 8 noticeable seizures during a 28-day period where they were recorded in a diary?
Does your child have any other significant genetic conditions that might cause their epilepsy?
Has your child had worsening seizures after taking a 'sodium channel blocker' medicine?

Answer every question to see your result.

What does participation involve?

If you decide to take part, your child will be monitored carefully throughout the study. This will involve regular visits to the clinic, where doctors and nurses will check their health, take blood samples, and perform other routine assessments. You will also need to keep a daily diary to record your child's seizures.

Your child will be given study medicine (either PRAX-562 or a placebo) to take for a period. The study will last several months, including an initial screening phase, a period of taking the medicine, and follow-up visits. The total duration will be explained in detail by the study team.

Throughout their participation, there will be opportunities to discuss any concerns or questions you have with the study team. You can always decide to stop taking part at any time.

Potential risks and benefits

Taking part in a clinical trial may offer potential benefits, such as access to a new medicine that isn't yet widely available, and helping advance medical knowledge for others with similar conditions. However, like all medicines, PRAX-562 may have side effects, and some tests might be uncomfortable. The study team will explain all known risks. You have the right to withdraw your child from the study at any time, for any reason, without affecting their future medical care.

Locations (8)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Praxis Research Site
Verified postcode
Atlanta, United States
Praxis Research Site
Verified postcode
Chicago, United States
Praxis Research Site
Verified postcode
Minneapolis, United States
Praxis Research Site
Verified postcode
Hackensack, United States
Praxis Research Site
Verified postcode
Tel Litwinsky, Israel
Praxis Research Site
Verified postcode
Madrid, Spain
Praxis Research Site
Verified postcode
Glasgow, United Kingdom
Praxis Research Site
Verified postcode
London, United Kingdom

Common questions

What is SCN2A or SCN8A encephalopathy?

These are types of severe epilepsy caused by changes in specific genes (SCN2A or SCN8A). They often lead to frequent and difficult-to-control seizures and can affect a child's development.

What is PRAX-562?

PRAX-562 is a new medicine being tested as a potential treatment for these specific types of epilepsy. It's hoped it might help reduce seizures.

Will my child definitely get the new medicine?

No, because this is a 'blinded' study, your child will either receive PRAX-562 or a 'placebo' (a dummy medicine) for a period. This is to fairly test the medicine's effects.

How long does the study last?

The study involves several stages, including screening, taking the study medicine, and follow-up visits. The total time your child would be involved will be explained by the study team.

Can I take my child out of the study if we change our mind?

Yes, you are free to withdraw your child from the study at any time, for any reason, without any impact on their usual medical care.