Condition: Pelizaeus-Merzbacher Disease · Sponsor: Ionis Pharmaceuticals, Inc.
The purpose of the study is to prospectively assess longitudinal changes in proteolipid protein 1 (PLP1) protein, disease-related biomarkers in cerebral spinal fluid (CSF) and blood, neuroimaging parameters relevant to Pelizaeus-Merzbacher disease (PMD) and longitudinal changes in performance on clinical, participant, and caregiver-reported outcome assessments to inform the development of therapies for PMD.
This description comes directly from the study's public registry record.
Ionis Pharmaceuticals · (844) 430-1848 · IonisPelizaeusMerzbacherStudy@clinicaltrialmedia.com
Always discuss trial participation with your own doctor first.
| Ionis Investigative Site | Atlanta, Georgia, United States | Recruiting |
| Ionis Investigative Site | Philadelphia, Pennsylvania, United States | Recruiting |
| Ionis Investigative Site | Clermont-Ferrand, France | Active Not Recruiting |
| Ionis Investigative Site | Le Kremlin-Bicêtre, France | Active Not Recruiting |
| Ionis Investigative Site | Göttingen, Germany | Active Not Recruiting |
| Ionis Investigative Site | Tel Aviv, Israel | Recruiting |
| Ionis Investigative Site | Milan, Italy | Recruiting |
| Ionis Investigative Site | Amsterdam, Netherlands | Recruiting |
| Ionis Investigative Site | Leeds, United Kingdom | Recruiting |
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Source record: clinicaltrials.gov/study/NCT05659901