A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

Conditions

• Dravet Syndrome

Eligibility Criteria

Sex

ALL

Age

6 Months - 17 Years

Healthy Volunteers

Not accepted

Interventions

• ETX101 — DRUG
  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 Details

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.

Key Dates

Start date

May 14, 2024

Status verified

Feb 2026

Primary completion

Jan 31, 2028

Completion

Jan 31, 2033

Study Design

Enrollment

47 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Part 1 (US Only)
  Part 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.
• Sham Comparator: Part 2
  Part 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.

Primary Outcome Measure

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). ]

Central Contacts

• Encoded Patient Advocacy
  +1 (650) 398-4301
  [email protected]

Locations (10)

Find similar trials in San Francisco, CA

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