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A Phase II, open-label, single arm basket trial to assess the safety, tolerability, and clinical activity of PLX-200, in Neuronal Ceroid Lipofuscinosis (CLN2, CLN3), Krabbe disease, and Sandhoff disease

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Trial Profile

A Phase II, open-label, single arm basket trial to assess the safety, tolerability, and clinical activity of PLX-200, in Neuronal Ceroid Lipofuscinosis (CLN2, CLN3), Krabbe disease, and Sandhoff disease

Status: Planning
Phase of Trial: Phase II

Latest Information Update: 26 Feb 2026

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At a glance

  • Drugs PLX 200 (Primary)
  • Indications Globoid cell leukodystrophy; Neuronal ceroid lipofuscinosis; Sandhoff disease
  • Focus Therapeutic Use
  • Acronyms SOTERIA
  • Sponsors Polaryx Therapeutics

Most Recent Events

  • 17 Feb 2026 According to Polaryx Therapeutics media release, Polaryx received a safe to proceed letter from the FDA in October 2025 and continues to actively work with its contract research organization (CRO) to initiate the trial in the first half of 2026.
  • 13 Feb 2026 New trial record
  • 03 Feb 2026 According to Polaryx Therapeutics media release, the company will be presenting a late-breaking abstract on new data for PLX-200, an investigational therapy for the treatment of Krabbe disease (globoid cell leukodystrophy) at the 22nd Annual WORLDSymposium, held February 2-6, 2026 in San Diego, Calif.

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