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Clinical Trials in the UK / NCT05419492
Recruiting Phase 1/2

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

NCT05419492 · tracked via the Priya Life Science UK tracker
Sponsor
Encoded Therapeutics
Phase
Phase 1/2
Started
2024-05-14
Last updated
2026-08-21

Condition(s) studied

Dravet Syndrome

Investigational drug(s) / intervention(s)

ETX101

ETX101: ETX101 is a non-replicating, recombinant adeno-associated viral vector serotype 9 (rAAV9) comprising a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A). ETX101 is intended as a one-time intracerebroventricular (ICV) administration.

Study 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

Sex
ALL
Min age
6 Months
Max age
17 Years
Healthy volunteers
No
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.

Primary outcome measure(s)

Trial sites (14)

FacilityCityRegionStatus
UCSF Benioff Children's Hospitals San Francisco California Recruiting
Colorado Children's Hospital Aurora Colorado Recruiting
Nicklaus Children's Hospital Miami Florida Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago Chicago Illinois Recruiting
Boston Children's Hospital Boston Massachusetts Not Yet Recruiting
Mott Children's Hospital Ann Arbor Michigan Recruiting
Mayo Clinic Rochester Minnesota Recruiting
Duke Children's Hospital & Health Center Durham North Carolina Recruiting
Nationwide Children's Hospital Columbus Ohio Not Yet Recruiting
Oregon Health and Science University (OSHU) Portland Oregon Recruiting
Cook Children's Medical Center Fort Worth Texas Recruiting
The Royal Children's Hospital Melbourne Australia Recruiting
Queen Elizabeth Hospital Glasgow United Kingdom Recruiting
Great Ormond Street Hospital London United Kingdom Not Yet Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05419492 on ClinicalTrials.gov ↗ ← All trials in the UK