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Clinical Trial 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.


Clinical Trial Description

This is a multi-center, non-randomized, non-interventional integrated prospective and retrospective study in up to 20 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). ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05659901
Study type Observational
Source Ionis Pharmaceuticals, Inc.
Contact Ionis Pharmaceuticals
Phone (844) 430-1848
Email IonisPelizaeusMerzbacherStudy@clinicaltrialmedia.com
Status Recruiting
Phase
Start date October 3, 2022
Completion date August 2025

See also
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Completed NCT01391637 - Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD) Phase 1
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Recruiting NCT06150716 - Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD) Phase 1