EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to \< 48 months. The study follows and open-label, dose-escalation design.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
5
ETX101 is composed of a non-replicating, recombinant adeno-associated viral serotype 9 (rAAV9) vector used to deliver a GABAergic regulatory element (reGABA) and an engineered transcription factor that increases transcription of the SCN1A gene (eTFSCN1A)
Queen Elizabeth Hospital
Glasgow, United Kingdom
Great Ormond Street Hospital
London, United Kingdom
Sheffield Children's Hospital
Sheffield, United Kingdom
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
Time frame: Baseline to Week 52
Change from Baseline in Vineland-3 subdomain GSVs at Week 52.
Time frame: Baseline to Week 52.
Change from Baseline in Bayley-4 subdomain GSVs at Week 52.
Time frame: 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: Baseline to Week 52.
Change from Baseline in Vineland-3 subdomain raw scores at Week 52.
Time frame: Baseline to Week 52.
Change from Baseline in Bayley-4 subdomain raw scores (excluding cognitive subdomain) at Week 52.
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Time frame: 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: 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: Baseline to Week 52.