Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
A Phase 1b Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease
Ionis Pharmaceuticals, Inc.
24 participants
Apr 10, 2024
INTERVENTIONAL
Conditions
Summary
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
Eligibility
Plain Language Summary
Simplified for easier understanding
This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.
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Interventions
Administered as intrathecal (IT) injection.
Locations(7)
View Full Details on ClinicalTrials.gov
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NCT06150716