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

RecruitingPhase 1, Phase 2Ages 6 Months - 17 YearsAllEncoded Therapeutics
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In plain words

What the trial is, and whether it could suit you

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 โ‰ฅ6 to \<36 months (Part 1A), aged โ‰ฅ48 months to \<18 years (Part 1B), and aged โ‰ฅ6 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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
Inclusion Criteria: * Participant must be aged between โ‰ฅ6 months and \<36 months in Part 1A, โ‰ฅ48 months and \<18 years in Part 1B, โ‰ฅ6 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.
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What it tests

  • ETX101
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Where

14 sites
UCSF Benioff Children's Hospitals
San Francisco, United States
Colorado Children's Hospital
Aurora, United States
Nicklaus Children's Hospital
Miami, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States
Mott Children's Hospital
Ann Arbor, United States
Mayo Clinic
Rochester, United States
NYU Langone Health
New York, United States
Duke Children's Hospital & Health Center
Durham, United States

โ€ฆand 6 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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