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
RECRUITINGColorado Children's Hospital
Aurora, Colorado, United States
NOT_YET_RECRUITINGPercent 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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Nicklaus Children's Hospital
Miami, Florida, United States
RECRUITINGAnn & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
RECRUITINGBoston Children's Hospital
Boston, Massachusetts, United States
NOT_YET_RECRUITINGOregon Health and Science University (OSHU)
Portland, Oregon, United States
RECRUITINGCook Children's Medical Center
Fort Worth, Texas, United States
RECRUITINGThe Royal Children's Hospital
Melbourne, Australia
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.