Clinical Trial

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

Recruiting Phase 1
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Summary
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
Protocol Amendment History 10 amendments
This ClinicalTrials.gov record has been amended 10 times since 2023-11-21; most recent amendment 2025-12-05.
Status change: Not Yet Recruiting → Recruiting 2024-04-12
Trial Details
NCT Number NCT06150716
Lead Sponsor Ionis Pharmaceuticals, Inc.
Conditions Pelizaeus-Merzbacher Disease
Enrollment 24 participants
Start Date 2024-04-10
Primary Completion 2028-06 (estimated)
Study Completion 2028-06 (estimated)
Updated on ClinicalTrials.gov 2025-12-12