New hope for children battling rare brain disease
NCT ID NCT06181136
Summary
This early-stage study is testing an experimental drug called DNL126 in children with Sanfilippo syndrome type A, a rare and devastating genetic disorder that causes progressive brain damage. Researchers will assess the drug's safety, how it moves through the body, and look for early signs that it might help reduce harmful substances in the brain and spinal fluid. The study will follow participants for up to four years to gather information.
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Contacts and locations
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Locations
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Baylor College of Medicine and Texas Children's Hospital
Houston, Texas, 77030, United States
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UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
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University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242, United States
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University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514, United States
Conditions
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