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

• Status: Not yet recruiting
• Trial end date: 2029-10-01
• Target enrollment: 0
• Participant gender: All

Summary

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 1 follows an open-label, dose-escalation design, and Part 2 is a randomized, double-blind, sham delayed-treatment control, dose-selection study.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Encoded Therapeutics

Criteria

Inclusion Criteria:

• Participant must have a predicted loss of function pathogenic or likely pathogenic SCN1A variant.

• Participant must have experienced their first convulsive 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.

Exclusion Criteria:

• 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 is currently taking or has taken antiseizure medications (ASMs) at a therapeutic dose that are contraindicated in Dravet syndrome, including sodium channel blockers.

• 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, including under an expanded access and/or compassionate use program.

• Participant has clinically significant underlying liver disease.