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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
47
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.
UCSF Benioff Children's Hospitals
San Francisco, California, United States
RECRUITINGNicklaus Children's Hospital
Miami, Florida, United States
NOT_YET_RECRUITINGAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
Percent change in monthly countable seizure frequency (MCSF) between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period.
Time frame: Between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period (defined as Week 5 to Week 52).
Change from Baseline in Bayley-4 cognitive subdomain raw score at Week 52 (Key Secondary Endpoint for Part 2).
Time frame: From Baseline to Week 52.
Change from Baseline in Vineland-3 subdomain GSVs at Week 52.
Time frame: From Baseline to Week 52.
Change from Baseline in Bayley-4 subdomain GSVs at Week 52.
Time frame: From Baseline to Week 52.
Proportion of participants achieving ≥ 75% reduction in MCSF between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period.
Time frame: Between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period (defined as Week 5 to Week 52).
Proportion of participants achieving ≥ 50% reduction in MCSF between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period.
Time frame: Between the Pre-Dosing Seizure Period and the Post-Dosing Assessment Period (defined as Week 5 to Week 52).
Change from Baseline in the Vineland-3 Adaptive Behavior Composite standard score at Week 52.
Time frame: From Baseline to Week 52.
Change from Baseline in Vineland-3 subdomain raw scores at Week 52.
Time frame: From Baseline to Week 52.
Change from Baseline in Bayley-4 subdomain raw scores (excluding cognitive subdomain) at Week 52.
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Cook Children's Medical Center
Fort Worth, Texas, United States
RECRUITINGThe Royal Children's Hospital
Melbourne, Australia
NOT_YET_RECRUITINGQueen Elizabeth Hospital
Glasgow, United Kingdom
NOT_YET_RECRUITINGGreat Ormond Street Hospital
London, United Kingdom
NOT_YET_RECRUITINGTime frame: From Baseline to Week 52.
Proportion of CGI-I responders, defined as participants with a CGI-I score of 1 (Very much improved) or 2 (Much improved), at Week 52.
Time frame: From Baseline to Week 52.
Proportion of CGI-S responders, defined as participants who either have a CGI-S score of 1 (Normal, not at all ill) or demonstrate a ≥2 point improvement from Baseline, at Week 52.
Time frame: From Baseline to Week 52.