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

NCT05659901 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 32

Last updated 2026-04-17

No results posted yet for this 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

Sponsors & Collaborators

  • Ionis Pharmaceuticals, Inc.

    lead INDUSTRY

Eligibility

Min Age
6 Months
Max Age
17 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-10-03
Primary Completion
2029-03-31
Completion
2029-03-31

Countries

  • United States
  • France
  • Germany
  • Israel
  • Italy
  • Netherlands
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05659901 on ClinicalTrials.gov