Evaluating a gene therapy for children with Dravet syndrome
WAYFINDER: a Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children with SCN1A-positive Dravet Syndrome
PHASE1; PHASE2 · Encoded Therapeutics · NCT06112275
This study is testing a new gene therapy for children with Dravet syndrome to see if it is safe and helps reduce their seizures.
Quick facts
| Phase | PHASE1; PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 4 (estimated) |
| Ages | 6 Months to 83 Months |
| Sex | All |
| Sponsor | Encoded Therapeutics (industry) |
| Locations | 1 site (Melbourne) |
| Trial ID | NCT06112275 on ClinicalTrials.gov |
What this trial studies
This clinical study aims to assess the safety and efficacy of ETX101, a gene therapy delivered via AAV9, in children diagnosed with SCN1A-positive Dravet syndrome. The study is designed as an open-label, dose-escalation trial, focusing on participants aged 6 to less than 84 months. It will evaluate how well the treatment works and monitor any potential side effects in this specific patient population.
Who should consider this trial
Good fit: Ideal candidates are children aged 6 months to under 7 years with a confirmed SCN1A mutation and a clinical diagnosis of Dravet syndrome.
Not a fit: Patients with other genetic mutations or significant comorbidities that could affect the typical presentation of Dravet syndrome may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could significantly improve seizure control and quality of life for children with Dravet syndrome.
How similar studies have performed: While gene therapies for epilepsy are emerging, this specific approach targeting SCN1A-positive Dravet syndrome is relatively novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Melbourne
- The Royal Children's Hospital — Melbourne, Australia (RECRUITING)
Study contacts
- Study coordinator: Encoded Patient Advocacy
- Email: patientadvocacy@encoded.com
- Phone: +1 (650) 398-4301
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Dravet Syndrome, Dravet, SCN1A, DEE, developmental and epileptic encephalopathy, SCN1A-positive, SCN1A+