ClinicaliQ Trial Snapshot
- Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease — Recruiting • NCT05659901.
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What This Trial Is Studying
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. Conditions: Pelizaeus-Merzbacher Disease Lead Sponsor: Ionis Pharmaceuticals, Inc. Planned Enrollment: 32 participants