Summary

Eligibility
for people ages 6 months to 47 months (full criteria)
Location
at UCSF
Dates
study started
completion around
Principal Investigator
by Joseph Sullivan (ucsf)
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) and aged ≥6 to <48 months (Part 2). 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 47 months

  • Participant must be aged between ≥6 months and <36 months in Part 1 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 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 accepting new patients
    San Francisco California 94158 United States
  • Cook Children's Medical Center accepting new patients
    Fort Worth Texas 76104 United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago not yet accepting patients
    Chicago Illinois 60611 United States

Lead Scientist at University of California Health

  • Joseph Sullivan (ucsf)
    Professor, Neurology, School of Medicine. Authored (or co-authored) 58 research publications

Details

Status
accepting new 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