New hope for rare childhood brain disease
NCT ID NCT06488924
Summary
This early-stage study is testing a new drug called JR-446 in children under 18 with a rare, serious genetic disorder called MPS IIIB (Sanfilippo syndrome type B). The main goals are to check if the drug is safe and to see how it affects the body. Researchers will enroll about 10 participants to gather initial data on safety and potential effects.
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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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