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NCT05419492PHASE1, PHASE2Recruiting

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

Encoded Therapeutics

Start Date

5/14/2024

Completion Date

1/1/2033

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 Criteria

Age Range: No minimum to 17 years

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.

Interventions

DRUG

ETX101

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Conditions

Dravet Syndrome

Locations

UCSF Benioff Children's Hospitals

San Francisco, California 94158

United States

Colorado Children's Hospital

Aurora, Colorado 80045

United States

Nicklaus Children's Hospital

Miami, Florida 33155

United States

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

Chicago, Illinois 60611

United States

Boston Children's Hospital

Boston, Massachusetts 02115

United States

Mott Children's Hospital

Ann Arbor, Michigan 48109

United States

Mayo Clinic

Rochester, Minnesota 55905

United States

Duke Children's Hospital & Health Center

Durham, North Carolina 27705

United States

Nationwide Children's Hospital

Columbus, Ohio 43205

United States

Oregon Health and Science University (OSHU)

Portland, Oregon 97239

United States

Cook Children's Medical Center

Fort Worth, Texas 76104

United States

The Royal Children's Hospital

Melbourne,

Australia

Queen Elizabeth Hospital

Glasgow, G51 4TF

United Kingdom

Great Ormond Street Hospital

London, WC1N3JH

United Kingdom