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

RECRUITINGPHASE1/PHASE2

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

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

NCT05419492•Encoded Therapeutics

View on ClinicalTrials.gov

Summary

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.

Eligibility

Age

0 - 17 years

Sex

ALL

Healthy Volunteers

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.

Locations (8)

UCSF Benioff Children's Hospitals

San Francisco, California, United States

Nicklaus Children's Hospital

Miami, Florida, United States

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

Chicago, Illinois, United States

Oregon Health and Science University (OSHU)

Portland, Oregon, United States

Cook Children's Medical Center

Fort Worth, Texas, United States

The Royal Children's Hospital

Melbourne, Australia

Queen Elizabeth Hospital

Glasgow, United Kingdom

Great Ormond Street Hospital

London, United Kingdom

Key Dates

Start Date

May 14, 2024

Primary Completion Date

January 1, 2028

Completion Date

January 1, 2033

Last Updated

February 24, 2026

Enrollment

ESTIMATED participants

Conditions

Dravet Syndrome

Interventions

ETX101

DRUG

Contacts

Encoded Patient Advocacy

CONTACT

+1 (650) 398-4301 patientadvocacy@encoded.com

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Dravet Syndrome

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: February 24, 2026Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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

Inclusion Criteria

Exclusion Criteria

A Study of EPX-100 (Clemizole Hydrochloride) in Participants With Dravet Syndrome

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