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

Recruitingn/aAges 6 Months - 17 YearsMaleIonis Pharmaceuticals, Inc.
NCT05659901 on ClinicalTrials.gov ↗
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In plain words

What the trial is, and whether it could suit you

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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📋 Eligibility rules (as published)
Inclusion Criteria: 1. Participant has a parent or caregiver capable of providing informed consent (signed and dated) and able to attend all scheduled study visits and provide feedback regarding the participant's symptoms and performance as described in the protocol and be able to comply with all study requirements 2. Participant has a diagnosis of Pelizaeus-Merzbacher Disease with genetic confirmation of PLP1 duplication 3. Male, 6 months-17 years old, inclusive, at the time of informed consent and phenotype consistent with classic PMD 4. No contraindications for lumbar punctures (LPs), blood draws, neuroimaging, sedation (if necessary) or other study procedures Exclusion Criteria: 1. Clinically significant abnormalities in medical history or physical examination 2. \> 2 copies of the PLP1 gene 3. Have any other conditions, which, in the opinion of the investigator would make the participant unsuitable for inclusion, or could interfere with the participant taking part in or completing the study
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What it tests

  • Not listed
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Where

10 sites
Ionis Investigative Site
Atlanta, United States
Ionis Investigative Site
Baltimore, United States
Ionis Investigative Site
Philadelphia, United States
Ionis Investigative Site
Clermont-Ferrand, France
Ionis Investigative Site
Le Kremlin-Bicêtre, France
Ionis Investigative Site
Göttingen, Germany
Ionis Investigative Site
Tel Aviv, Israel
Ionis Investigative Site
Milan, Italy

…and 2 more on ClinicalTrials.gov.

🤝 Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible — always decide together with your own doctor.
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