One-Time gene therapy injection aims to halt devastating childhood disease
NCT ID NCT02716246
Summary
This study is testing a one-time gene therapy called UX111 for children with Sanfilippo Syndrome Type A, a rare and severe genetic disorder. The therapy involves a single intravenous injection designed to deliver a working copy of a missing gene, with the goal of slowing or stopping the disease's progression in the brain. The trial is enrolling up to 36 children, primarily aged 6 months to 5 years, to evaluate the treatment's safety and whether it reduces harmful substances and helps preserve development.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital of Pittsburgh
COMPLETEDPittsburgh, Pennsylvania, 15224, United States
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Hospital Clínico Universitario de Santiago
RECRUITINGSantiago de Compostela, 15706, Spain
Contact Email: •••••@•••••
Contact Email: •••••@•••••
Contact
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Nationwide Children's Hospital
COMPLETEDColumbus, Ohio, 43205, United States
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Vall d'Hebron Barcelona Hospital Campus
RECRUITINGBarcelona, Barcelona, 08035, Spain
Contact Email: •••••@•••••
Contact
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Women's and Children's Hospital
COMPLETEDNorth Adelaide, South Australia, 5006, Australia
Conditions
Explore the condition pages connected to this study.