NCT05419492 A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
| NCT ID | NCT05419492 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | Encoded Therapeutics |
| Condition | Dravet Syndrome |
| Study Type | INTERVENTIONAL |
| Enrollment | 113 participants |
| Start Date | 2024-05-14 |
| Primary Completion | 2028-06 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 113 participants in total. It began in 2024-05-14 with a primary completion date of 2028-06.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 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.
Eligibility Criteria
Inclusion Criteria: * 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 1 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 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.
Contact & Investigator
Salvador Rico, M.D., Ph.D
STUDY DIRECTOR
Encoded Therapeutics
Frequently Asked Questions
Who can join the NCT05419492 clinical trial?
This trial is open to participants of all sexes, aged 6 Months or older, up to 17 Years, studying Dravet Syndrome. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05419492 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05419492 currently recruiting?
Yes, NCT05419492 is actively recruiting participants. Contact the research team at patientadvocacy@encoded.com for enrollment information.
Where is the NCT05419492 trial being conducted?
This trial is being conducted at San Francisco, United States, Aurora, United States, Miami, United States, Chicago, United States and 10 additional locations.
Who is sponsoring the NCT05419492 clinical trial?
NCT05419492 is sponsored by Encoded Therapeutics. The principal investigator is Salvador Rico, M.D., Ph.D at Encoded Therapeutics. The trial plans to enroll 113 participants.