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Gene Therapy Trial Report

Summary

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


NCTID NCT05419492 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Dravet Syndrome
Disease Ontology Term DOID:0080422
Compound Name ETX101
Compound Description rAAV9-reGABA-eTFSCN1A
Sponsor Encoded Therapeutics
Funder Type Industry
Recruitment Status
Enrollment Count 47 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant SCN1A
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intracerebroventricular
Drug Product Type Viral vector
Target Tissue/Cell GABAergic inhibitory interneurons
Delivery System Viral transduction
Vector Type AAV9
Editor Type none
Dose 1 Undisclosed low dose
Dose 2 Undisclosed high dose

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2022-06-10
Completion Date 2033-01
Last Update 2026-02-24

Participation Criteria


Eligible Age 6 Months - 17 Years
Standard Ages Child
Sexes Eligible for Study ALL
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 8
Locations United States,United Kingdom,Australia

Regulatory Information


Has US IND True
FDA Designations 🔄 Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
Recent Updates Received Breakthrough Designation from FDA in January 2026

Resources/Links