First human trial launches for rare childhood brain disease
NCT ID NCT06150716
Summary
This study is the first to test a new drug called ION356 in children with Pelizaeus-Merzbacher disease (PMD), a rare genetic disorder that affects the brain and nerves. The main goal is to check if the drug is safe and how the body processes it when given as a spinal injection. About 24 boys, aged 2 to 17, with a confirmed genetic diagnosis will participate and receive the drug for nearly three years to monitor its effects.
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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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
RECRUITINGSalt Lake City, Utah, 84113, United States
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Ionis Investigative Site
RECRUITINGLe Kremlin-Bicêtre, 94270, France
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Ionis Investigative Site
RECRUITINGGöttingen, 37075, Germany
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Ionis Investigative Site
RECRUITINGTokyo, 187-8551, Japan
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Ionis Investigative Site
RECRUITINGAmsterdam, 1105AZ, Netherlands
Conditions
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