Skip to content
Bored Studies
RecruitingPhase 1/Phase 2

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

Encoded Therapeutics · NCT05419492

View Study & Apply (opens in a new tab)

Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 6 mo–17 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 1, Phase 2
Target enrollment
113
US sites recruiting
10
Study start
May 14, 2024
Record last updated
October 23, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: ETX101

Conditions: Dravet Syndrome

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full 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.

Recruiting US locations

  • UCSF Benioff Children's Hospitals

    San Francisco, CA 94158

    Site contact: Adam Numis, MD · Adam.numis@ucsf.edu

  • Colorado Children's Hospital

    Aurora, CO 80045

    Site contact: Kallie Dock · 720-777-5322 · Kallie.dock@childrenscolorado.org

  • Nicklaus Children's Hospital

    Miami, FL 33155

    Site contact: Matt Lallas · 305-662-8330 · Matt.Lallas@nicklaushealth.org

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, IL 60611

    Site contact: Jazmine Ibarra · 312-227-0967 · jazibarra@luriechildrens.org

  • Mott Children's Hospital

    Ann Arbor, MI 48109

    Site contact: Stephanie Rau · 734-232-8474 · shatchew@med.umich.edu

  • Mayo Clinic

    Rochester, MN 55905

    Site contact: Bridget Neja · 507-266-9150 · neja.bridget@mayo.edu

  • Duke Children's Hospital & Health Center

    Durham, NC 27705

    Site contact: Makenzi Buchanan · 919-613-1232 · Makenzi.hayes@duke.edu

  • Nationwide Children's Hospital

    Columbus, OH 43205

    Site contact: Kaitlyn Brown · 614-722-2286 · Kaitlyn.brown2@nationwidechildrens.org

  • Oregon Health and Science University (OHSU)

    Portland, OR 97239

    Site contact: Emily Stein · steine@ohsu.edu

  • Cook Children's Medical Center

    Fort Worth, TX 76104

    Site contact: Neurology Epilepsy Research · (682) 885-2844 · neuroepilepsyresearch@cookchildrens.org

Study contacts (from the official record)

  • Encoded Patient Advocacy · +1 (650) 398-4301 · patientadvocacy@encoded.com
All neurology studies in California →
A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in… (NCT05419492) | Bored Studies