A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Primary outcome measure(s)
- Percent change in monthly countable seizure frequency (MCSF) between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period. — Between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period (defined as Week 5 to Week 52).
Trial sites (14)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
| Mott Children's Hospital | Ann Arbor | Michigan | Recruiting |
| Mayo Clinic | Rochester | Minnesota | Recruiting |
| NYU Langone Health | New York | New York | Not Yet Recruiting |
| Duke Children's Hospital & Health Center | Durham | North Carolina | Recruiting |
| Nationwide Children's Hospital | Columbus | Ohio | Recruiting |
| Oregon Health and Science University (OHSU) | 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 | Recruiting |
More Encoded Therapeutics trials in the UK
Other trials for the same condition
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