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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)

Recruiting
36 - 83 years of age
Both
Phase 1/2

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Overview

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 36 to <84 months (3 to <7 years). The study follows an open-label, dose-escalation design.

Eligibility

Inclusion Criteria:

  • Participant must have a predicted loss of function pathogenic or likely pathogenic SCN1A variant.
  • Participant must have experienced their first convulsive 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 details

Dravet Syndrome

NCT06112275

Encoded Therapeutics

21 February 2024

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