Summary

Eligibility
for people ages 6 months to 35 months (full criteria)
Location
at San Francisco, California and other locations
Dates
study started
completion around
Principal Investigator
by Joseph Sullivan, MD
Headshot of Joseph Sullivan
Joseph Sullivan

Description

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.

Official Title

ENDEAVOR: A Clinical Study to Evaluate the Safety and Efficacy of ETX101, an AAV9-Delivered Gene Therapy in Infants and Children With SCN1A-Positive Dravet Syndrome

Keywords

Dravet Syndrome, Dravet, SCN1A, DEE, developmental and epileptic encephalopathy, SCN1A-positive, SCN1A+, Myoclonic Epilepsies, Syndrome, ETX101

Eligibility

You can join if…

Open to people ages 6 months to 35 months

  • 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.

You CAN'T join 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 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.

Locations

  • UCSF Benioff Children's Hospitals
    San Francisco California 94158 United States
  • Cook Children's Medical Center
    Fort Worth Texas 76104 United States

Lead Scientist at UCSF

  • Joseph Sullivan, MD
    Professor, Neurology, School of Medicine. Authored (or co-authored) 53 research publications

Details

Status
not yet accepting patients
Start Date
Completion Date
(estimated)
Sponsor
Encoded Therapeutics
ID
NCT05419492
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
Expecting 22 study participants
Last Updated