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)
- Registration Number
- NCT06112275
- Lead Sponsor
- Encoded Therapeutics
- 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, dose-escalation design.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 4
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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Cohort A ETX101 Cohort A will evaluate ETX101 dose level 1. Cohort B ETX101 Cohort B will evaluate ETX101 dose level 2. Cohort C ETX101 Cohort C will evaluate ETX101 dose level 3. Cohort D ETX101 Cohort D will evaluate ETX101 dose level 4.
- Primary Outcome Measures
Name Time Method 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. 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 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. Day 1 through Study Completion, an average of 5 years Proportion of participants free from episodes of prolonged seizures and/or status epilepticus at Week 52. Week 5 through Week 52
- Secondary Outcome Measures
Name Time Method Change from Baseline in the Vineland-Third Edition Expressive Communication raw score at Week 52. Baseline through Week 52. Higher scores correspond to better outcomes. Proportion of participants with ≥ 90% reduction in monthly countable seizure frequency (MCSF) from Baseline at Week 52. 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)
Trial Locations
- Locations (1)
The Royal Children's Hospital
🇦🇺Melbourne, Australia