PMD Clinical Study

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

Study Overview

Brief Summary

The primary purpose of this study is to evaluate the safety and tolerability of ION356.

Detailed Description

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.

Orbit Study recently added a new recruitment site to the ORBIT study in the United States (The Children’s Hospital of Philadelphia – CHOP). You can find more information about the study and newly added sites here. As a reminder, any individuals interested in participating should speak with their doctor or contact our team at +1 844-387-9520 or [email protected] to learn more about sites and enrollment.  

To learn more visit: https://www.clinicaltrials.gov/study/NCT06150716?term=orbit%20pmd&rank=1#collaborators-and-investigators