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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 🔄 2026-04-16

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 🔄 10
Locations United States,United Kingdom,Australia

Regulatory Information


Has US IND True
FDA Designations 🔄 Breakthrough Therapy, Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy (RMAT)
Recent Updates Announced in May 2026 that the first patient in the pivotal trial has been dosed, initial data from Part 1B is expected in 4Q26

Resources/Links