Status:
ACTIVE_NOT_RECRUITING
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)
Lead Sponsor:
Encoded Therapeutics
Conditions:
Dravet Syndrome
Eligibility:
All Genders
6-83 years
Phase:
PHASE1
PHASE2
Brief 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, do...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
February 28 2024
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2030
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT06112275
Start Date
February 28 2024
End Date
September 1 2030
Last Update
November 14 2025
Active Locations (1)
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1
The Royal Children's Hospital
Melbourne, Australia