A Clinical Study to Evaluate the Safety and Efficacy of ETX101 in Infants and Children With SCN1A-Positive Dravet Syndrome
Start Date
5/14/2024
Completion Date
1/1/2033
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 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.
Eligibility Criteria
Age Range: No minimum to 17 years
Interventions
ETX101
Conditions
Locations
UCSF Benioff Children's Hospitals
San Francisco, California 94158
United States
Colorado Children's Hospital
Aurora, Colorado 80045
United States
Nicklaus Children's Hospital
Miami, Florida 33155
United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United States
Boston Children's Hospital
Boston, Massachusetts 02115
United States
Mott Children's Hospital
Ann Arbor, Michigan 48109
United States
Mayo Clinic
Rochester, Minnesota 55905
United States
Duke Children's Hospital & Health Center
Durham, North Carolina 27705
United States
Nationwide Children's Hospital
Columbus, Ohio 43205
United States
Oregon Health and Science University (OSHU)
Portland, Oregon 97239
United States
Cook Children's Medical Center
Fort Worth, Texas 76104
United States
The Royal Children's Hospital
Melbourne,
Australia
Queen Elizabeth Hospital
Glasgow, G51 4TF
United Kingdom
Great Ormond Street Hospital
London, WC1N3JH
United Kingdom