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TrialConditionsStatusPhaseUpdatedMore infoNCT ID
Long-term Follow-Up for RGX-121Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Death, Death, Sudden, Infant Death, Neurobehavioral Manifestations, Neurologic Manifestations, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Skin and Connective Tissue Diseases, Sudden Infant Death, X-linked disease, connective tissue disorder, cutaneous mucinosis, hereditary disease, inborn carbohydrate metabolic disorder, inborn errors of metabolism, inherited neurodegenerative disorder, intellectual disability, lysosomal storage disease, metabolic disease, mucopolysaccharidosis, mucopolysaccharidosis type 2, nervous system disorderActive, not recruitingNot stated2026-08-19NCT04597385

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OrganizationsLinked trials
REGENXBIO Inc.14

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Research sitesCityCountryLinked trials
Hospital de Clinicas de Porto AlegrePorto AlegreBrazil25
Saint Peter's Healthcare SystemNew BrunswichUnited States1
The Children's Hospital of Philadelphia (CHOP)PhiladelphiaUnited States7
University of California, San Francisco (UCSF)- Benioff Children's HospitalSan FranciscoUnited States1