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A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

RecruitingPhase 1/2

ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Infants and Children With SCN1A-Positive Dravet Syndrome

Who can join

6 Months – 17 Years · All sexes

Full eligibility criteria
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.

About the study

ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to \<36 months (Part 1A), aged ≥48 months to \<18 years (Part 1B), and aged ≥6 to \<48 months (Part 2). Part 1A follows an open-label, dose-escalation design, Part 1B follows an open-label design, and Part 2 is a randomized, double-blind, sham delayed-treatment control study.

What is being tested

Sponsor: Encoded Therapeutics · Participants: 47 · Started: May 14, 2024

Contact the study team

Official record on ClinicalTrials.gov — NCT05419492

Locations in the U.S.

CaliforniaUCSF Benioff Children's Hospitals, San Francisco
ColoradoColorado Children's Hospital, Aurora
FloridaNicklaus Children's Hospital, Miami
IllinoisAnn & Robert H. Lurie Children's Hospital of Chicago, Chicago
MassachusettsBoston Children's Hospital, Boston (Not yet recruiting)
MichiganMott Children's Hospital, Ann Arbor
MinnesotaMayo Clinic, Rochester
North CarolinaDuke Children's Hospital & Health Center, Durham
OhioNationwide Children's Hospital, Columbus
OregonOregon Health and Science University (OSHU), Portland
TexasCook Children's Medical Center, Fort Worth

Conditions

From ClinicalTrials.gov, data retrieved Oct 2, 2026. Each study sets its own eligibility; the study team decides who can join.