EXPEDITION: 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 all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY
EXPEDITION is a Phase 1/2 study in the UK to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet Syndrome aged 6 to \< 48 months. The study follows and open-label, dose-escalation design.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 6 months
Maximum Age: 3
Healthy Volunteers: f
View:
• Participant has a predicted loss of function pathogenic or likely pathogenic SCN1A variant
• Participant must have experienced their first seizure between the age of 3 and 15 months
• Participant must have a clinical diagnosis of Dravet syndrome or the treating clinician must have high clinical suspicion of a diagnosis of Dravet syndrome
• Participant is receiving at least one prophylactic antiseizure medication
Locations
Other Locations
United Kingdom
Queen Elizabeth Hospital
RECRUITING
Glasgow
Great Ormond Street Hospital
RECRUITING
London
Sheffield Children's Hospital
RECRUITING
Sheffield
Contact Information
Primary
Encoded Patient Advocacy
patientadvocacy@encoded.com
+1 (650) 398-4301
Time Frame
Start Date: 2024-05-09
Estimated Completion Date: 2030-09
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