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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

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Trial Profile

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

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 18 Jun 2025

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At a glance

  • Drugs ETX 101 (Primary) ; ETX 101 (Primary)
  • Indications Dravet syndrome; Seizures
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms ENDEAVOR
  • Sponsors Encoded Therapeutics

Most Recent Events

  • 13 Feb 2025 According to an Encoded Therapeutics media release, Completion of additional dose levels and reporting of preliminary safety and efficacy data are planned for 2H25.
  • 02 Dec 2024 Protocol was amended to add patients aged >-6 to <48 months (Part 2), patient maximum age changed from 35 months to 47 Months, hence there were few changes in the primary endpoint and treatment arm.
  • 15 Apr 2024 Planned End Date changed from 1 May 2031 to 1 Apr 2031.

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