Actively Recruiting
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Led by Encoded Therapeutics · Updated on 2026-04-16
47
Participants Needed
10
Research Sites
450 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
ENDEAVOR is a Phase 1/2, 2-part, multicenter study to evaluate the safety and efficacy of ETX101 in participants with SCN1A-positive Dravet syndrome aged ≥6 to \<36 months (Part 1A), aged ≥48 months to \<18 years (Part 1B), and aged ≥6 to \<48 months (Part 2). Part 1A follows an open-label, dose-escalation design, Part 1B follows an open-label design, and Part 2 is a randomized, double-blind, sham delayed-treatment control study.
CONDITIONS
Official Title
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant aged between 6 months and under 36 months in Part 1A, between 48 months and under 18 years in Part 1B, or between 6 months and under 48 months in Part 2
- Participant has a predicted loss of function pathogenic or likely pathogenic SCN1A variant
- Participant experienced their first seizure between 3 and 15 months of age
- Participant has a clinical diagnosis or strong clinical suspicion of Dravet syndrome
- Participant is receiving at least one preventive antiseizure medication
You will not qualify if you...
- Participant has another genetic mutation or clinical condition that could affect typical Dravet syndrome features
- Participant has known central nervous system structural or vascular abnormalities seen on brain MRI or CT
- Participant has conditions interfering with cerebrospinal fluid distribution or an existing ventriculoperitoneal shunt
- Participant received sodium channel blockers during the pre-dosing seizure period
- Participant has been seizure-free for 4 consecutive weeks within 90 days before consent
- Participant has previously received gene or cell therapy
- Participant is currently enrolled in another clinical trial or receiving investigational treatment
- Participant has significant liver disease
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 10 locations
1
UCSF Benioff Children's Hospitals
San Francisco, California, United States, 94158
Actively Recruiting
2
Colorado Children's Hospital
Aurora, Colorado, United States, 80045
Not Yet Recruiting
3
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
4
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
5
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Not Yet Recruiting
6
Oregon Health and Science University (OSHU)
Portland, Oregon, United States, 97239
Actively Recruiting
7
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Actively Recruiting
8
The Royal Children's Hospital
Melbourne, Australia
Actively Recruiting
9
Queen Elizabeth Hospital
Glasgow, United Kingdom, G51 4TF
Not Yet Recruiting
10
Great Ormond Street Hospital
London, United Kingdom, WC1N3JH
Not Yet Recruiting
Research Team
E
Encoded Patient Advocacy
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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