A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome

Recruiting now · Phase 1/Phase 2

Conditions studied: Dravet Syndrome

In brief

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.

Key facts

Study ID

NCT05419492

Run by

Encoded Therapeutics

People needed

47

Starts

2024-05-14

Expected to finish

2033-01-01

Last updated by the study team

2026-07-29

Who can join

Age: 1 and older, up to 17. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Participant must be aged between ≥6 months and <36 months in Part 1A, ≥48 months and <18 years in Part 1B, ≥6 months and <48 months in Part 2.
• 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.

You may not qualify if…

• Participant has another genetic mutation or clinical comorbidity which could potentially confound the typical Dravet phenotype.
• Participant has a known central nervous system structural and/or vascular abnormality (indicated by an MRI or CT scan of the brain).
• Participant has an abnormality that may interfere with CSF distribution and/or has an existing ventriculoperitoneal shunt.
• Participant has received sodium channel blockers during the Pre-Dosing Seizure Period.
• Participant has experienced seizure freedom for a period of 4 consecutive weeks within the 90-day period prior to informed consent.
• Participant has previously received gene or cell therapy.
• Participant is currently enrolled in a clinical trial or receiving an investigational therapy.
• Participant has clinically significant underlying liver disease.

Where it is running

• Cook Children's Medical Center — Fort Worth, Texas, United States (enrolling)
• The Royal Children's Hospital — Melbourne, Australia (enrolling)
• Queen Elizabeth Hospital — Glasgow, United Kingdom (enrolling)
• UCSF Benioff Children's Hospitals — San Francisco, California, United States (enrolling)
• Colorado Children's Hospital — Aurora, Colorado, United States (enrolling)
• Mott Children's Hospital — Ann Arbor, Michigan, United States (enrolling)
• Mayo Clinic — Rochester, Minnesota, United States (enrolling)
• Nicklaus Children's Hospital — Miami, Florida, United States (enrolling)
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States (enrolling)
• Oregon Health and Science University (OSHU) — Portland, Oregon, United States (enrolling)
• Great Ormond Street Hospital — London, United Kingdom
• Boston Children's Hospital — Boston, Massachusetts, United States
• Duke Children's Hospital & Health Center — Durham, North Carolina, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States

Full record on ClinicalTrials.gov

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