Recruiting

Testing a new gene therapy for rare childhood seizure disorder

Plain-English translation of NCT05419492 on ClinicalTrials.gov ↗ · Source last updated July 2026 · Translation generated July 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

What is this trial?

ENDEAVOR is a clinical trial testing , a gene therapy designed to treat infants and young children with Dravet syndrome, a rare and severe genetic seizure disorder. The study will evaluate whether this treatment is safe and whether it can reduce seizures in children ages 6 months to 18 years. This medication works by delivering a corrected gene into the body to help reduce seizures at their source.

Dravet syndrome is a severe, lifelong seizure disorder that begins in infancy and is difficult to treat with current medications alone. This trial exists to see if this new gene therapy approach can offer a better treatment option for children who have not responded well to available seizure medications.

Do you qualify?

You likely qualify if…

you are between 6 months and 36 months old (Part 1A), OR between 4 and 18 years old (Part 1B), OR between 6 months and 4 years old (Part 2)
you have been diagnosed with or have a high suspicion of Dravet syndrome caused by a specific genetic mutation called SCN1A
you experienced your first seizure between 3 and 15 months of age
you are currently taking at least one anti-seizure medication
you have not had seizure freedom for 4 or more weeks in the 90 days before enrolling

You likely don't qualify if…

you have another genetic mutation or medical condition that could complicate your Dravet syndrome diagnosis
you have a known structural abnormality or blood vessel problem in your brain
you have a brain shunt or condition that would interfere with how fluid moves in your brain
you have serious liver disease
you have previously received gene therapy or cell therapy, or are currently in another clinical trial

What participation looks like

Participation involves either receiving or, in Part 2, possibly receiving a sham procedure with delayed treatment (meaning you would receive the real treatment later). Participants will have multiple visits for safety monitoring, seizure tracking, and testing over many months. The study uses a dose-escalation approach in the first part, meaning the first groups receive lower doses to ensure safety, and later groups may receive higher doses if the treatment appears safe.

AI-generated summary from trial data · Jul 10, 2026 · Not medical advice

Trial locations(14 sites)

United States

UCSF Benioff Children's Hospitals, San Francisco, California

Colorado Children's Hospital, Aurora, Colorado

Nicklaus Children's Hospital, Miami, Florida

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

Boston Children's Hospital, Boston, Massachusetts