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New hope for babies with rare seizure disorder: drug trial targets SCN2A gene

NCT ID NCT07019922

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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 04, 2026 · Updated 2 times

Summary

This study tests a medicine called elsunersen in 40 children with a rare, severe form of epilepsy caused by changes in the SCN2A gene. The goal is to see if the drug can safely reduce how often seizures happen over 24 weeks. Children must have started having seizures before 3 months old and have at least 4 seizures per month to join.

Why investors are watching

Praxis Precision Medicines is running a Phase 3 trial of elsunersen in 40 children with early onset SCN2A developmental and epileptic encephalopathy, a severe genetic seizure disorder. For a micro-cap company with few other late-stage assets, this readout could determine whether it has a viable commercial product or faces a major setback.

If it works: A positive result could support a regulatory submission for elsunersen, giving Praxis its first approved therapy and a revenue source. That outcome would validate the company's approach to treating this rare disease.

If it fails: The trial could fail to show meaningful seizure control or safety problems, which would likely end the program and leave Praxis without a clear path forward. Trials in rare pediatric epilepsy often fail, so investors should treat success as uncertain.

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 40 people

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

Started

Aug 2025

Expected to finish

Dec 2028

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

1 day to 18 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 Gain of Function SCN2A variant confirmed through genetic testing. * Has onset of seizures prior to 3 months of age. * Seizure frequency of 4 or more countable motor seizures per 28-day during the Baseline Observation Period. Exclusion Criteria: * Has any clinically significant or known pathogenic genetic variant other than in the SCN2A gene, or a genetic variant that may explain or contribute to the participant's epilepsy and/or developmental disorder. * Has bone, spine (eg, kyphosis, scoliosis), bleeding, or other disorder. * Has received any 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.

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Conditions

The condition(s) this trial relates to.

developmental and epileptic encephalopathy developmental and epileptic encephalopathy, 11 epilepsy visual epilepsy

As listed by the trial registrant

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

Epileptic encephalopathy SCN2A encephalopathy

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    8 sites in 3 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Praxis Research Site

    RECRUITING

    San Diego, California, 92123, United States

  • Praxis Research Site

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Praxis Research Site

    RECRUITING

    Chicago, Illinois, 60612, United States

  • Praxis Research Site

    RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Praxis Research Site

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Praxis Research Site

    RECRUITING

    Memphis, Tennessee, 38105, United States

  • Praxis Research Site

    WITHDRAWN

    Porto Alegre, 90035, Brazil

  • Praxis Research Site

    WITHDRAWN

    São Paulo, 05403, Brazil

  • Praxis Research Site

    RECRUITING

    Bonn, Bonn, 53127, Germany

  • Praxis Research Site

    RECRUITING

    Rome, 00165, Italy

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