New study aims to unlock secrets of rare brain disease in children
NCT ID NCT05659901
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This observational study is following 32 boys aged 6 months to 17 years with Pelizaeus-Merzbacher disease (PMD), a rare genetic disorder affecting the brain's white matter. Researchers are measuring biomarkers in spinal fluid and blood, brain scans, and motor and cognitive skills over time. The goal is to better understand how PMD progresses, which could help develop future treatments.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
What this could lead to
If successful, this study could identify key markers of PMD progression, helping researchers design better treatments for this rare disease.
What could go wrong
This is an observational study, not a treatment trial. It may not directly benefit participants, and results may not lead to immediate therapies.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ionis Investigative Site
RECRUITINGAtlanta, Georgia, 30342, United States
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Ionis Investigative Site
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Ionis Investigative Site
ACTIVE_NOT_RECRUITINGClermont-Ferrand, France
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Ionis Investigative Site
ACTIVE_NOT_RECRUITINGLe Kremlin-Bicêtre, 94270, France
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Ionis Investigative Site
ACTIVE_NOT_RECRUITINGGöttingen, Germany
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Ionis Investigative Site
RECRUITINGTel Aviv, 6423906, Israel
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Ionis Investigative Site
RECRUITINGMilan, 20154, Italy
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Ionis Investigative Site
RECRUITINGAmsterdam, 1081 HV, Netherlands
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Ionis Investigative Site
RECRUITINGLeeds, LS1 3EX, United Kingdom