A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Public ClinicalTrials.gov record NCT05419492. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Infants and Children With SCN1A-Positive Dravet Syndrome
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT05419492
- Recruitment status
- Recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 47 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 6 Months to 17 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- May 13, 2024
- Primary completion
- Dec 31, 2027
- Completion
- Dec 31, 2032
- Last update posted
- Aug 20, 2026
2024 – 2033
United States locations
- U.S. sites
- 11
- U.S. states
- 11
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| UCSF Benioff Children's Hospitals | San Francisco | California | 94158 | Recruiting |
| Colorado Children's Hospital | Aurora | Colorado | 80045 | Recruiting |
| Nicklaus Children's Hospital | Miami | Florida | 33155 | Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | Recruiting |
| Boston Children's Hospital | Boston | Massachusetts | 02115 | Not yet recruiting |
| Mott Children's Hospital | Ann Arbor | Michigan | 48109 | Recruiting |
| Mayo Clinic | Rochester | Minnesota | 55905 | Recruiting |
| Duke Children's Hospital & Health Center | Durham | North Carolina | 27705 | Recruiting |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | Not yet recruiting |
| Oregon Health and Science University (OSHU) | Portland | Oregon | 97239 | Recruiting |
| Cook Children's Medical Center | Fort Worth | Texas | 76104 | Recruiting |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05419492, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 20, 2026 · Synced Sep 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05419492 live on ClinicalTrials.gov.