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A Phase 1/2, Open-Label Clinical Study to Evaluate Safety, Tolerability, and Efficacy of NGN-401 in Subjects With Rett Syndrome

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Trial Profile

A Phase 1/2, Open-Label Clinical Study to Evaluate Safety, Tolerability, and Efficacy of NGN-401 in Subjects With Rett Syndrome

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 29 May 2025

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At a glance

  • Drugs NGN 401 (Primary)
  • Indications Rett syndrome
  • Focus Adverse reactions; First in man
  • Sponsors Neurogene Inc

Most Recent Events

  • 23 May 2025 Planned number of patients changed from 16 to 14.
  • 16 May 2025 According to a Neurogene media release, company announced an oral presentation at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting describing a monitoring and treatment algorithm intended to reverse the rare, severe hyperinflammatory syndrome hemophagocytic lymphohistiocytosis (HLH) that has been associated with systemic exposure to high doses of adeno-associated virus (AAV) gene therapy
  • 18 Nov 2024 According to a Neurogene media release, The U.S. Food and Drug Administration (FDA) has completed a review of the safety data for NGN-401 and has allowed company to proceed with this trial using the 1E15 vg dose. And Following a treatment-related serious adverse event (SAE) in the third participant dosed with 3E15 vg, company paused further use of the 3E15 vg dose and does not plan to enroll any further participants at the 3E15 vg dose.

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