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Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)

Orbit Study: Testing ION356 for safety in Pelizaeus Merzbacher Disease patients.

Recruiting
2-17 years
Male
Phase 1
  • Simplified description (AI rewritten)
  • Technical description (Original)

This study checks if a new treatment, **ION356**, is safe for kids with a rare brain condition called *Pelizaeus-Merzbacher Disease (PMD)*. PMD happens when there's an extra copy of the *PLP1 gene*. The study lasts for about 3 years, split into two parts: the first part of 48 weeks and the second part of 109 weeks. During the study, kids will get doses of ION356. To be in the study, kids need to be boys aged 2-17 with PMD, and their parents must agree to participate. The study requires several visits to the study center. Kids can't join if they have other major health problems or have taken certain medicines recently. Kids who have had brain surgery or issues with MRI (a type of brain scan) can't join either. The study ensures safety with regular check-ups. It's important for parents to discuss with their doctor if this study is a good fit for their child.

  • The study lasts for about 3 years with regular visits.
  • Safe participation requires no other major health issues.
  • Eligible boys must be 2-17 years old with confirmed PMD.
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Study details
    Pelizaeus-Merzbacher Disease

NCT06150716

Ionis Pharmaceuticals, Inc.

4 May 2025

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