A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome (Australia Only)

November 13, 2025 updated by: Encoded Therapeutics

WAYFINDER: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children With SCN1A-positive Dravet Syndrome

WAYFINDER is a Phase 1/2 study in Australia to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged 6 to <84 months. The study follows an open-label, dose-escalation design.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

4

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Melbourne, Australia
        • The Royal Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Participant must have a predicted loss of function pathogenic or likely pathogenic SCN1A variant.
  • Participant must have experienced their first seizure between the ages of 3 and 15 months.
  • Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have a high clinical suspicion of a diagnosis of Dravet syndrome.
  • Participant is receiving at least one prophylactic antiseizure medication.

Exclusion Criteria:

  • Participant has another genetic mutation or clinical comorbidity which could potentially confound the typical Dravet phenotype.
  • Participant has a known central nervous system structural and/or vascular abnormality (indicated by an MRI or CT scan of the brain).
  • Participant has an abnormality that may interfere with CSF distribution and/or has an existing ventriculoperitoneal shunt.
  • Participant is currently taking or has taken antiseizure medications (ASMs) at a therapeutic dose that are contraindicated in Dravet syndrome, including sodium channel blockers.
  • Participant has experienced seizure freedom for a period of 4 consecutive weeks within the 6-month period prior to informed consent.
  • Participant has previously received gene or cell therapy.
  • Participant is currently enrolled in a clinical trial or receiving an investigational therapy.
  • Participant has clinically significant underlying liver disease.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort A
Cohort A will evaluate ETX101 dose level 1.
Drug: ETX101
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).
Experimental: Cohort B
Cohort B will evaluate ETX101 dose level 2.
Drug: ETX101
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).
Experimental: Cohort C
Cohort C will evaluate ETX101 dose level 3.
Drug: ETX101
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).
Experimental: Cohort D
Cohort D will evaluate ETX101 dose level 4.
Drug: ETX101
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).

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Proportion of participants experiencing any treatment-emergent adverse events (AEs), serious adverse events (SAEs), related AEs, AEs with severity Grade ≥ 3, AEs resulting in study discontinuation, and AEs with a fatal outcome.
Time Frame: Day 1 through Study Completion, an average of 5 years
Day 1 through Study Completion, an average of 5 years
Percent change from Baseline in monthly countable seizure frequency (MCSF) at Week 52, with countable seizures defined as generalized tonic-clonic/clonic, focal motor with clearly observable clinical signs, tonic bilateral, and atonic seizures.
Time Frame: Between the 8-week baseline period and the 48-week post-dosing assessment period (defined as Week 5 to Week 52 following administration of ETX101)
Between the 8-week baseline period and the 48-week post-dosing assessment period (defined as Week 5 to Week 52 following administration of ETX101)
Proportion of participants free from episodes of prolonged seizures and/or status epilepticus at Week 52.
Time Frame: Week 5 through Week 52
Week 5 through Week 52

Secondary Outcome Measures

Outcome Measure
Time Frame
Proportion of participants with ≥ 90% reduction in monthly countable seizure frequency (MCSF) from Baseline at Week 52.
Time Frame: Between the 8-week baseline period and the 48-week post-dosing assessment period (defined as Week 5 to Week 52 following administration of ETX101)
Between the 8-week baseline period and the 48-week post-dosing assessment period (defined as Week 5 to Week 52 following administration of ETX101)
Change from Baseline in the Vineland-Third Edition Expressive Communication raw score at Week 52.
Time Frame: Baseline through Week 52. Higher scores correspond to better outcomes.
Baseline through Week 52. Higher scores correspond to better outcomes.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Encoded Therapeutics

Investigators

  • Study Director: Salvador Rico, M.D., Ph.D, Encoded Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 28, 2024

Primary Completion (Estimated)

September 1, 2030

Study Completion (Estimated)

September 1, 2030

Study Registration Dates

First Submitted

October 25, 2023

First Submitted That Met QC Criteria

October 30, 2023

First Posted (Actual)

November 1, 2023

Study Record Updates

Last Update Posted (Estimated)

November 14, 2025

Last Update Submitted That Met QC Criteria

November 13, 2025

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ETX-DS-004

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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