● Recruiting

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

Condition: Pelizaeus-Merzbacher Disease · Sponsor: Ionis Pharmaceuticals, Inc.

PhasePhase 1

Planned participants24

Who can joinMale, 2 Years to 17 Years

Healthy volunteersNo

About this study

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

This description comes directly from the study's public registry record.

Talk to the study team

Ionis Pharmaceuticals, Inc. · (844) 387-9520 · IonisPelizaeusMerzbacherStudy2@clinicaltrialmedia.com

Always discuss trial participation with your own doctor first.

Locations (7)

Ionis Investigative Site	Atlanta, Georgia, United States	Recruiting
Ionis Investigative Site	Philadelphia, Pennsylvania, United States	Recruiting
Ionis Investigative Site	Salt Lake City, Utah, United States	Recruiting
Ionis Investigative Site	Le Kremlin-Bicêtre, France	Recruiting
Ionis Investigative Site	Göttingen, Germany	Recruiting
Ionis Investigative Site	Tokyo, Japan	Recruiting
Ionis Investigative Site	Amsterdam, Netherlands	Recruiting

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Source record: clinicaltrials.gov/study/NCT06150716