Recruiting studies: Sanfilippo Syndrome Type A

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Phase 1 · 1 location(s) · Joanne Kurtzberg, MD · NCT02254863 Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
Phase N/A · 2 location(s) · Phoenix Nest · NCT05825131 Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Phase 2/Phase 3 · 5 location(s) · Ultragenyx Pharmaceutical Inc · NCT02716246 Phase 1 Study of GC1130A in Patients With Sanfilippo Syndrome Type A (MPS IIIA)
Phase 1 · 5 location(s) · GC Biopharma Corp · NCT06567769 An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambrox
Phase 2/Phase 3 · 1 location(s) · Ozlem Goker-Alpan · NCT06614894 Longitudinal Study of Neurodegenerative Disorders
Phase N/A · 1 location(s) · University of Pittsburgh · NCT03333200

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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.