Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
Start Date
10/3/2022
Completion Date
3/1/2029
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).
Eligibility Criteria
Age Range: No minimum to 17 years
Conditions
Locations
Ionis Investigative Site
Atlanta, Georgia 30342
United States
Ionis Investigative Site
Philadelphia, Pennsylvania 19104
United States
Ionis Investigative Site
Clermont-Ferrand,
France
Ionis Investigative Site
Le Kremlin-Bicêtre, 94270
France
Ionis Investigative Site
Göttingen,
Germany
Ionis Investigative Site
Tel Aviv, 6423906
Israel
Ionis Investigative Site
Milan, 20154
Italy
Ionis Investigative Site
Amsterdam, 1081 HV
Netherlands
Ionis Investigative Site
Leeds, LS1 3EX
United Kingdom