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Gene therapy trial aims to tame severe childhood epilepsy

NCT ID NCT05419492

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 21, 2026 · Updated 11 times

Summary

This study tests a gene therapy called ETX101 for children with Dravet syndrome, a severe form of epilepsy caused by a genetic change. The treatment uses a harmless virus to deliver a working copy of the gene to help control seizures. The trial includes infants and children up to 18 years old and will measure how well the therapy reduces seizure frequency and improves development over time.

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

About 47 people

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

Started

May 2024

Expected to finish

Jan 2033

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 months to 17 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: * Participant must be aged between ≥6 months and \<36 months in Part 1A, ≥48 months and \<18 years in Part 1B, ≥6 months and \<48 months in Part 2. * Participant must have a predicted loss of function pathogenic or likely pathogenic SCN1A variant. * Participant must have experienced their first seizure between the ages of 3 and 15 months. * Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have a 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 has received sodium channel blockers during the Pre-Dosing Seizure Period. * 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 generalized epilepsy with febrile seizures plus, type 2

As listed by the trial registrant

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

Dravet syndrome

How to take part

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

  1. The places running it

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

  2. 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 ↗

  3. 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

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Boston Children's Hospital

    NOT_YET_RECRUITING

    Boston, Massachusetts, 02115, United States

  • Colorado Children's Hospital

    RECRUITING

    Aurora, Colorado, 80045, United States

  • Cook Children's Medical Center

    RECRUITING

    Fort Worth, Texas, 76104, United States

  • Duke Children's Hospital & Health Center

    RECRUITING

    Durham, North Carolina, 27705, United States

  • Great Ormond Street Hospital

    NOT_YET_RECRUITING

    London, WC1N3JH, United Kingdom

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55905, United States

  • Mott Children's Hospital

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • Nationwide Children's Hospital

    NOT_YET_RECRUITING

    Columbus, Ohio, 43205, United States

  • Nicklaus Children's Hospital

    RECRUITING

    Miami, Florida, 33155, United States

  • Oregon Health and Science University (OSHU)

    RECRUITING

    Portland, Oregon, 97239, United States

  • Queen Elizabeth Hospital

    RECRUITING

    Glasgow, G51 4TF, United Kingdom

  • The Royal Children's Hospital

    RECRUITING

    Melbourne, Australia

  • UCSF Benioff Children's Hospitals

    RECRUITING

    San Francisco, California, 94158, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.