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

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.

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Purpose of Study

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.

Eligibility

Age:
6 Months - 17
Sex:
Female
Accepting Volunteers:
YES
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Conditions:

Status:

RECRUITING

Study Type:

OBSERVATIONAL

Clinical Trials ID

NCT05659901

Read Detailed Summary:

Start Date / End Date:

10 2022 - 03 2029

Enrollment:

32

Full Details:

https://clinicaltrials.gov/ct2/show/NCT05659901

Study Locations Map: