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New hope for kids with rare epilepsy: drug trial targets seizure control

NCT ID NCT07010471

Disease control Sponsor: Praxis Precision Medicines Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Aug 05, 2026 · Updated 2 times

Summary

This study tests an investigational drug called relutrigine in 160 children with a severe form of epilepsy called developmental and epileptic encephalopathy (DEE). The goal is to see if the drug can safely reduce monthly seizure frequency compared to a placebo. After the initial blinded phase, all participants can receive the drug in an open-label extension.

Why investors are watching

Praxis Precision Medicines is running a Phase 3 trial of relutrigine in 160 people with developmental and epileptic encephalopathies, a group of severe childhood epilepsy disorders. For a micro-cap company, this readout is the main test of whether its lead drug works, and the result shapes whether the program can move toward regulatory review.

If it works: If relutrigine controls seizures better than placebo, the company could use the data to support a regulatory filing and to seek a partner or funding for the open-label extension.

If it fails: If the trial misses its goal or faces delays, the company has few other late-stage assets to fall back on. Most Phase 3 trials fail, and a miss would set the program back.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 160 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2025

Expected to finish

Nov 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Has a documented diagnosis of a developmental and epileptic encephalopathy. * Onset of seizures \<12 years old. * Has a weight \>7 kg at the time of signing consent/assent. Exclusion Criteria: * Has a history of left bundle branch block, arrhythmias, Brugada syndrome, congenital heart disease, familial short QT syndrome, or family history of sudden death or ventricular arrhythmias, including idiopathic ventricular fibrillation. * Had 2 or more episodes of convulsive status epilepticus requiring hospitalization and intubation in the 6 months prior to Screening. * Has an abnormal ECG reading, including a QT interval corrected for heart rate using Bazett's method (QTcB) \<350 and \>450 ms (males), or \<360 and \>460 ms (females) at Screening and/or on Day 1. * Any nerve stimulation must have been placed at least 3 months prior to Screening with at least 1 month of stable settings prior to Screening. * Has received any other experimental or investigational drug, device, or other therapy within 30 days or 5 half-lives (whichever is longer) prior to Screening, including any prior use of gene therapy. * Is currently pregnant or breastfeeding or is planning to become pregnant during the clinical trial or within 5 half-lives of the last study drug dose.

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Conditions

The condition(s) this trial relates to.

developmental and epileptic encephalopathy developmental and epileptic encephalopathy, 1 epilepsy infantile epileptic-dyskinetic encephalopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Developmental and epileptic encephalopathy 1

Contacts and locations

Locations

  • Praxis Research Site

    La Jolla, California, 92037, United States

  • Praxis Research Site

    Gulf Breeze, Florida, 32561, United States

  • Praxis Research Site

    Chevy Chase, Maryland, 20815, United States

  • Praxis Research Site

    Roseville, Minnesota, 55113, United States

  • Praxis Research Site

    Hackensack, New Jersey, 07601, United States

  • Praxis Research Site

    Philadelphia, Pennsylvania, 19104, United States

  • Praxis Research Site

    Heidelberg, Victoria, 3084, Australia

  • Praxis Research Site

    Rio Grande, Do Sul, 90610, Brazil

  • Praxis Research Site

    Paraná, 80250, Brazil

  • Praxis Research Site

    Rio de Janeiro, 20231, Brazil

  • Praxis Research Site

    São Paulo, 15090, Brazil

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