WAYFINDER: a Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Children with SCN1A-positive Dravet Syndrome
Status: Recruiting
Location: See location...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 6 months
Maximum Age: 6
Healthy Volunteers: f
View:
• 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.
Locations
Other Locations
Australia
The Royal Children's Hospital
RECRUITING
Melbourne
Contact Information
Primary
Encoded Patient Advocacy
patientadvocacy@encoded.com
+1 (650) 398-4301
Time Frame
Start Date: 2024-02-28
Estimated Completion Date: 2030-06
Participants
Target number of participants: 4
Treatments
Experimental: Cohort A
Cohort A will evaluate ETX101 dose level 1.
Experimental: Cohort B
Cohort B will evaluate ETX101 dose level 2.
Experimental: Cohort C
Cohort C will evaluate ETX101 dose level 3.
Experimental: Cohort D
Cohort D will evaluate ETX101 dose level 4.
Related Therapeutic Areas
Sponsors
Leads: Encoded Therapeutics