Actively Recruiting
ENDEAVOR: Evaluating ETX101 Gene Therapy Safety and Efficacy in Infants and Children With SCN1A-Positive Dravet Syndrome
Led by Encoded Therapeutics · Updated on 2026-06-03
47
Participants Needed
13
Research Sites
261 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating ETX101, an investigational gene therapy delivered via AAV9, in infants and children with Dravet syndrome caused by SCN1A gene mutations. This study, called ENDEAVOR, includes participants from 6 months up to less than 18 years old and aims to assess the safety and effects of ETX101 on seizure frequency and developmental outcomes. The trial includes multiple parts with different age groups and study designs to understand its impact comprehensively. The study has three parts: Part 1A is an open-label dose-escalation phase for children aged 6 to less than 36 months, testing up to four dose levels of ETX101. Part 1B is an open-label phase for participants aged 48 months to less than 18 years, evaluating a single dose level. Part 2 is a randomized, double-blind, sham delayed-treatment control study for children aged 6 to less than 48 months, where participants are assigned in a 2:1 ratio to receive ETX101 or a sham procedure with delayed treatment. During participation, children will be monitored for changes in seizure frequency from before dosing to 52 weeks after treatment. Researchers will also assess cognitive and adaptive behavior development using standard scales like Bayley-4 and Vineland-3. Safety and efficacy will be closely observed through clinical evaluations, with follow-up lasting up to a year post-dosing to measure treatment outcomes and monitor health.
CONDITIONS
Brief 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 is between 6 months and less than 36 months old for Part 1A, between 48 months and less than 18 years old for Part 1B, or between 6 months and less than 48 months old for Part 2.
- Participant has a loss of function pathogenic or likely pathogenic SCN1A gene variant.
- Participant experienced their first seizure between 3 and 15 months of age.
- Participant has a clinical diagnosis of Dravet syndrome or a treating clinician suspects 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 symptoms.
- Participant has a known brain structural or vascular abnormality as shown by MRI or CT scan.
- Participant has any abnormality interfering with cerebrospinal fluid distribution or has a ventriculoperitoneal shunt.
- Participant has used 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 an investigational therapy.
- Participant has significant liver disease.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Week 0 (day of administration)
Participants receive a one-time administration of the ETX101 gene therapy or a sham procedure, depending on randomization.
1 treatment visit (in-person)
Duration - Week 5 to Week 52
Participants are monitored for safety and efficacy outcomes, including seizure frequency and developmental assessments.
Regular visits through Week 52
Trial Site Locations
Total: 13 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
Mott Children's Hospital
Ann Arbor, Michigan, United States, 48109
Not Yet Recruiting
7
Mayo Clinic
Rochester, Minnesota, United States, 55905
Not Yet Recruiting
8
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Not Yet Recruiting
9
Oregon Health and Science University (OSHU)
Portland, Oregon, United States, 97239
Actively Recruiting
10
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Actively Recruiting
11
The Royal Children's Hospital
Melbourne, Australia
Actively Recruiting
12
Queen Elizabeth Hospital
Glasgow, United Kingdom, G51 4TF
Actively Recruiting
13
Great Ormond Street Hospital
London, United Kingdom, WC1N3JH
Not Yet Recruiting
Research Team
E
Encoded Patient Advocacy
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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