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

Rocket Study: Exploring disease progression in Pelizaeus-Merzbacher Disease patients.

Recruiting
6-17 years
Male
Phase N/A
  • Simplified description (AI rewritten)
  • Technical description (Original)
Rocket Study: Understanding Pelizaeus-Merzbacher Disease The Rocket Study is focused on learning more about Pelizaeus-Merzbacher Disease (PMD), a rare genetic disorder affecting the brain. Researchers are studying changes in proteins and other disease markers in fluids like blood and cerebral spinal fluid (CSF), as well as brain imaging and patient performance. These findings will help develop treatments for PMD. This study is for up to 32 children and teens ages 6 months to 17 years who have PMD and can safely undergo procedures like blood tests, CSF collection, and brain scans. Participants will be part of the study for about 26 months. They need a caregiver who can attend visits and provide feedback. Key Points:
  • The study lasts for about 26 months with multiple visits.
  • No new treatments are given, only observations and tests.
  • Participants should not have more than 2 copies of the PLP1 gene.
Note: Participants must have a confirmed PMD diagnosis and meet specific health requirements. This helps ensure safety and reliability in study results.
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Study details
    Pelizaeus-Merzbacher Disease

NCT05659901

Ionis Pharmaceuticals, Inc.

17 October 2026

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