Clinical Trial

Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease

Recruiting
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Summary
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.
Protocol Amendment History 11 amendments
This ClinicalTrials.gov record has been amended 11 times since 2022-12-13; most recent amendment 2026-04-16.
Trial Details
NCT Number NCT05659901
Lead Sponsor Ionis Pharmaceuticals, Inc.
Conditions Pelizaeus-Merzbacher Disease
Enrollment 32 participants
Start Date 2022-10-03
Primary Completion 2029-03 (estimated)
Study Completion 2029-03 (estimated)
Updated on ClinicalTrials.gov 2026-04-17