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Gene therapy trial aims to tame seizures in kids with rare epilepsy

NCT ID NCT06283212

Disease control Sponsor: Encoded Therapeutics 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This early-stage study tests a gene therapy called ETX101 in 5 children with Dravet syndrome, a severe form of epilepsy. The therapy delivers a gene that helps calm overactive brain cells. Researchers will check if it safely reduces seizure frequency and improves thinking and daily skills over a year.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ETX101 (a gene therapy that boosts SCN1A gene activity in the brain)
What this could lead to
If it works, this could point toward a treatment that reduces seizures and improves developmental outcomes in children with Dravet syndrome.
What could go wrong
This is a very early, small trial (only 5 children) testing safety and dosing. Gene therapies can have side effects like immune reactions, and it may not work for everyone.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

5 people

The number who actually took part.

Started

May 2024

Expected to finish

Oct 2030

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

6 to 47 months

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: * Participant has a predicted loss of function pathogenic or likely pathogenic SCN1A variant * Participant must have experienced their first seizure between the age of 3 and 15 months * Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have high clinical suspicion of a diagnosis of Dravet syndrome * Participant is receiving at least one prophylactic antiseizure medication Exclusion Criteria: * Participant has another genetic mutation or clinical comorbidity which could potentially confound the typical Dravet phenotype * Participant has a known central nervous system structural and/or vascular abnormality (indicated by an MRI or CT scan of the brain). * Participant has an abnormality that may interfere with CSF distribution and/or has an existing ventriculoperitoneal shunt. * Participant is currently taking or has taken antiseizure medications (ASMs) at a therapeutic dose that are contraindicated in Dravet syndrome, including sodium channel blockers. * Participant has experienced seizure freedom for a period of 4 consecutive weeks within the 90-day period prior to informed consent. * Participant has previously received gene or cell therapy. * Participant is currently enrolled in a clinical trial or receiving an investigational therapy. * Participant has clinically significant underlying liver disease.

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Conditions

The condition(s) this trial relates to.

Dravet syndrome Epilepsies, Myoclonic

As listed by the trial registrant

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

Dravet syndrome

Contacts and locations

Locations

  • Great Ormond Street Hospital

    London, WC1N3JH, United Kingdom

  • Queen Elizabeth Hospital

    Glasgow, G51 4TF, United Kingdom

  • Sheffield Children's Hospital

    Sheffield, S10 2TH, United Kingdom

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