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A Phase 3, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacodynamics of RGX 121 in Pediatric Participants With Neuronopathic MPS II (Hunter Syndrome)

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Trial Profile

A Phase 3, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacodynamics of RGX 121 in Pediatric Participants With Neuronopathic MPS II (Hunter Syndrome)

Status: Recruiting
Phase of Trial: Phase III

Latest Information Update: 10 Dec 2025

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At a glance

  • Drugs Clemidsogene lanparvovec (Primary)
  • Indications Mucopolysaccharidosis II
  • Focus Adverse reactions; Registrational; Therapeutic Use
  • Sponsors REGENXBIO

Most Recent Events

  • 27 Nov 2025 New trial record
  • 26 Nov 2025 Status changed from not yet recruiting to recruiting.

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