New hope for kids with rare brain disease: drug trial launches
NCT ID NCT06488924
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 27 times
Summary
This study tests a new drug called JR-446 in 10 children under 18 with Sanfilippo B syndrome (MPS IIIB), a rare genetic disease that damages the brain. The main goals are to see if the drug is safe and to measure its effects on the body. Researchers will also check how the drug moves through the body and if it can help slow the disease.
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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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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Hiroshima University Hospital
RECRUITINGHiroshima, Japan
Contact Phone: •••-•••-••••
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National Center for Child Health and Development
RECRUITINGTokyo, Japan
Contact Phone: •••-•••-••••
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University of the Ryukyus Hospital
RECRUITINGOkinawa, Japan
Contact Phone: •••-•••-••••
Conditions
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