New drug trial offers hope for rare brain disease in boys
NCT ID NCT06150716
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage study tests a new medicine called ION356 in 24 boys aged 2 to 17 with Pelizaeus-Merzbacher disease, a rare genetic brain disorder. The main goal is to check if the drug is safe and how the body processes it. The drug is given through a spinal injection to target the brain directly.
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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
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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