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

Description

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.

Conditions

Dravet Syndrome

Talk to us

Study Overview

On this page

Study Details

Study overview

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.

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

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

Condition
Dravet Syndrome
Intervention / Treatment

-

Contacts and Locations

San Francisco

UCSF Benioff Children's Hospitals, San Francisco, California, United States, 94158

Chicago

Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States, 60611

Fort Worth

Cook Children's Medical Center, Fort Worth, Texas, United States, 76104

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * 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.
  • * 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.

Ages Eligible for Study

6 Months to 47 Months

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Encoded Therapeutics,

Salvador Rico, M.D., Ph.D, STUDY_DIRECTOR, Encoded Therapeutics

Study Record Dates

2031-04