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

Study 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.

Conditions

Pelizaeus-Merzbacher Disease

Interventions / Drugs

View on ClinicalTrials.gov ↗

Sponsor

IONS

Ionis Pharmaceuticals

Antisense Therapeutics

B68/100

Study Details

Enrollment32 participants

Primary Completion—

Lead SponsorIonis Pharmaceuticals, Inc.

Data Retrieved2026-04-11 04:17:53.139399+00:00

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