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A Phase 1b Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease

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Trial Profile

A Phase 1b Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 09 May 2025

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At a glance

  • Drugs ION 356 (Primary)
  • Indications Pelizaeus-Merzbacher disease
  • Focus Adverse reactions; Therapeutic Use
  • Acronyms Orbit Study
  • Sponsors Ionis Pharmaceuticals

Most Recent Events

  • 12 Apr 2024 Status changed from not yet recruiting to recruiting.
  • 18 Dec 2023 Planned initiation date changed from 1 Dec 2023 to 1 Mar 2024.
  • 21 Nov 2023 Status changed from planning to not yet recruiting.

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