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ETX101

Phase 1

Dravet Syndrome | Small molecule | Neurology |cbdMD, Inc.|Last Updated: Aug 21, 2026

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindSHAM_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment47

FDA Designations

No designations recorded

Clinical trial landscape

ETX101 · 1 trial · 1 indication

Phase 1 1
NCT05419492A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet SyndromeDravet Syndrome
RECRUITING47 Analytics
PHASE1RECRUITING
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Dravet Syndrome
NCT0541949247
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Study Endpoints

Primary Endpoints

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).

Secondary Endpoints

Change from Baseline in Bayley-4 cognitive subdomain raw score at Week 52 (Key Secondary Endpoint for Part 2).
From Baseline to Week 52.
Change from Baseline in Vineland-3 subdomain GSVs at Week 52.
From Baseline to Week 52.
Change from Baseline in Bayley-4 subdomain GSVs at Week 52.
From Baseline to Week 52.
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Part 1 (US Only)EXPERIMENTALPart 1A will follow an open-label, rule-based, dose-escalation design and will evaluate up to 4 dose levels of ETX101. Part 1B will follow an open-label design and will evaluate 1 dose level of ETX101.
Part 2SHAM_COMPARATORPart 2 will follow a double-blind (up through Week 52), randomized, sham delayed-treatment control design. There will be 2 cohorts in Part 2. A single dose level of ETX101 will be evaluated in Part 2 and participants will be randomized 2:1 to study treatment or sham procedure with delayed treatment.

Interventions

NameTypeDescription
ETX101DRUGETX101 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.
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Eligibility Criteria

Age Range6 Months to 17 Years
SexALL
Healthy VolunteersNo
Study Sites14

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 experie...

Countries:United StatesAustraliaUnited Kingdom
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