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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)

NCT ID: NCT06112275

Last Updated: 2025-11-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-02-28

Study Completion Date

2030-09-30

Brief Summary

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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.

Detailed Description

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Conditions

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Dravet Syndrome

Keywords

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Dravet SCN1A DEE developmental and epileptic encephalopathy Dravet Syndrome SCN1A-positive SCN1A+

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort A

Cohort A will evaluate ETX101 dose level 1.

Group Type EXPERIMENTAL

ETX101

Intervention Type DRUG

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).

Cohort B

Cohort B will evaluate ETX101 dose level 2.

Group Type EXPERIMENTAL

ETX101

Intervention Type DRUG

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).

Cohort C

Cohort C will evaluate ETX101 dose level 3.

Group Type EXPERIMENTAL

ETX101

Intervention Type DRUG

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).

Cohort D

Cohort D will evaluate ETX101 dose level 4.

Group Type EXPERIMENTAL

ETX101

Intervention Type DRUG

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).

Interventions

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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).

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

6 Months

Maximum Eligible Age

83 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Encoded Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Salvador Rico, M.D., Ph.D

Role: STUDY_DIRECTOR

Encoded Therapeutics

Locations

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The Royal Children's Hospital

Melbourne, , Australia

Site Status

Countries

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Australia

Other Identifiers

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ETX-DS-004

Identifier Type: -

Identifier Source: org_study_id