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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.

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

Orbit Study is a research project to test the safety of a medicine called ION356 for children with Pelizaeus-Merzbacher Disease (PMD), a rare disorder that affects the brain. The study has two parts. The first part lasts 48 weeks where participants get increasing doses of ION356. After this, they move to the second part, which lasts 109 weeks, to continue receiving the medicine. PMD is linked to a problem with the PLP1 gene, and the study needs boys aged 2 to 17 years with this condition. Participants must be able to visit the study center and follow the study rules. However, if a child has had certain treatments or surgeries recently, they might not be able to join.

  • Duration: The study lasts for a total of 157 weeks (about 3 years).
  • Visits: Participants need to travel to the study center for check-ups.
  • Risks: There are checks to ensure safety, but there may be risks involved.
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Study details
    Pelizaeus-Merzbacher Disease

NCT06150716

Ionis Pharmaceuticals, Inc.

25 November 2024

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