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

Part of paid clinical trials in Atlanta, Georgia.

Sponsor
Ionis Pharmaceuticals, Inc.
Study ID
NCT05659901
Status
Recruiting
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Conditions

  • Pelizaeus-Merzbacher Disease

Eligibility Criteria

Sex
MALE
Age
6 Months - 17 Years
Healthy Volunteers
Not accepted

Study Details

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.

Key Dates

Start date
Oct 3, 2022
Status verified
Apr 2026
Primary completion
Mar 31, 2029
Completion
Mar 31, 2029

Study Design

Enrollment
32 participants (estimated)

Arms

  • Arm: Pelizaeus-Merzbacher Disease Participants
    Participants will undergo CSF collection and neuroimaging procedures, up to Week 106 as a part of prospective study. Each participant's medical and family history data will be collected retrospectively from available medical notes and charts, from birth up to the end of the study period (up to 26 months).

Primary Outcome Measure

Assess Longitudinal Changes in Fluid Biomarkers [ Time Frame: Up to 26 months ]

Central Contacts

Locations (2)

FacilityCityStateZIPSite coordinators
Ionis Investigative SiteAtlantaGeorgia30342-
Ionis Investigative SitePhiladelphiaPennsylvania19104-

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By research site
Ionis Investigative Site· Atlanta, GAIonis Investigative Site· Philadelphia, PA

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