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)

Last updated: February 25, 2025
Sponsor: Encoded Therapeutics
Overall Status: Active - Recruiting

Phase

1/2

Condition

Unverricht-lundborg Syndrome

Dravet Syndrome

Seizure Disorders (Pediatric)

Treatment

ETX101

Clinical Study ID

NCT06112275
ETX-DS-004
  • Ages 6-83
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Participant must have a predicted loss of function pathogenic or likely pathogenicSCN1A variant.

  • Participant must have experienced their first seizure between the ages of 3 and 15months.

  • Participant must have a clinical diagnosis of Dravet syndrome or the treatingclinician must have a high clinical suspicion of a diagnosis of Dravet syndrome.

  • Participant is receiving at least one prophylactic antiseizure medication.

Exclusion

Exclusion Criteria:

  • Participant has another genetic mutation or clinical comorbidity which couldpotentially confound the typical Dravet phenotype.

  • Participant has a known central nervous system structural and/or vascularabnormality (indicated by an MRI or CT scan of the brain).

  • Participant has an abnormality that may interfere with CSF distribution and/or hasan existing ventriculoperitoneal shunt.

  • Participant is currently taking or has taken antiseizure medications (ASMs) at atherapeutic dose that are contraindicated in Dravet syndrome, including sodiumchannel blockers.

  • Participant has experienced seizure freedom for a period of 4 consecutive weekswithin 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 aninvestigational therapy.

  • Participant has clinically significant underlying liver disease.

Study Design

Total Participants: 4
Treatment Group(s): 1
Primary Treatment: ETX101
Phase: 1/2
Study Start date:
February 28, 2024
Estimated Completion Date:
June 30, 2030

Connect with a study center

  • The Royal Children's Hospital

    Melbourne,
    Australia

    Active - Recruiting

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