Pelizaeus-Merzbacher Disease Clinical Trial
Official title:
A Phase 1b Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease
The primary purpose of this study is to evaluate the safety and tolerability of ION356.
This is a Phase 1b, open-label multiple-ascending dose (MAD) study of ION356 in approximately 24 pediatric participants with Pelizaeus-Merzbacher Disease and genetic confirmation of proteolipid protein 1 (PLP1) gene duplication. The study will have 2 parts: a 48-week multiple-ascending dose (MAD) part followed by an open-label long-term extension (LTE) part of 109 weeks. Eligible participants during the MAD portion of the study will receive doses of ION356 and upon completion will seamlessly transition to the open-label LTE to receive doses of ION356. ;
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