NCT05659901

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

Integrated Prospective and Retrospective Observational Study to Characterize Biomarkers and Disease Progression in Patients With Pelizaeus-Merzbacher Disease

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 32 (estimated)
• Sponsor: Ionis Pharmaceuticals, Inc. · Industry
• Sex: Male
• Age: 6 Months – 17 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is a multi-center, non-randomized, non-interventional integrated prospective and retrospective study in up to 32 participants with PMD who can undergo general anesthesia or conscious sedation (if necessary) to collect fluid biomarkers (CSF and blood), neuroimaging, and clinical assessments to be used in support of the development of therapies for PMD. The study duration for each participant will be approximately 26 months (Week 106).

Conditions

• Pelizaeus-Merzbacher Disease

Timeline

• Start date: 2022-10-03
• Primary completion: 2029-03-01
• Completion: 2029-03-01
• First posted: 2022-12-21
• Last updated: 2026-04-17

Locations

9 sites across 7 countries: United States, France, Germany, Israel, Italy, Netherlands, United Kingdom