Page last updated: July 17, 2026 For web accessibility options: Click/tap the floating blue icon on the right.Study Summary This is a REMOTE prospective observational study of participants with Sanfilippo Syndrome Type IIIC (MPS IIIC). Patients’ functional abilities...
Clinical Trial
Study of Cannabidiol in Sanfilippo Syndrome | MPS IIIA, IIIB, IIIC, and IIID | Phase II-III | Lundquist
Page last updated: March 31, 2026 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary Study: “Study of Epidiolex (cannabidiol) in Sanfilippo Syndrome” Trial Listing: Read this...
Ambroxol Hydrochloride | MPS IIIA, IIIB, IIIC, IIID | Phase II-III | Lysosomal & Rare Disorders Research & Treatment Center
Page last updated: August 20, 2025 For web accessibility options: Click/tap the floating blue icon on the right.Trial Information Study title: An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol...
JR-446 Enzyme Replacement | MPS IIIB | Phase I-II | MEDIPAL/JCR
Page last updated: August 13, 2026 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Trial Summary “JR-446 is a novel drug developed based on the J-Brain Cargo technology, which has been clinically...
Natural History Study of Participants With Sanfilippo Syndrome Type IIIC | MPS IIIC | Observational Study | Hospices Civils De Lyon
Page last updated: May 14, 2026 For web accessibility options: Click/tap the floating blue icon on the right.Study Summary In this multi-center, natural history study of subjects with Sanfilippo syndrome type C (MPS IIIC), patients will be monitored over a 2-year...
GC1130A Enzyme Replacement | MPS IIIA | Phase I | GC Biopharma
Page last updated: July 29, 2025 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary Study: Phase 1 Study of GC1130A in Pediatric Patients With Sanfilippo Syndrome Type A (MPS...
DNL126 Enzyme Replacement | MPS IIIA | Phase I-II | Denali
Page last updated: February 6, 2026 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Clinical Trial Summary Study: “A Phase 1/2, Multicenter, Open-Label Study to Evaluate the Safety, Tolerability,...
JR-441 Enzyme Replacement | MPS IIIA | Phase I-II | JCR
Page last updated: August 30, 2024 For web accessibility options: Click/tap the floating blue icon on the right. Trial Information Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA A Phase I/ II, open-label, randomized, 2-arm study, designed...
A Natural History Study of Patients With Sanfilippo Disease(s) (MPS3) | MPS IIIA, IIIB, IIIC, IIID | Observational Study | Lysosomal Center
Page last updated: January 31, 2023 For web accessibility options: Click/tap the floating blue icon on the right.Trial Information Lysosomal and Rare Disorders Research and Treatment Center, Inc. is conducting a natural history study of patients with Sanfilippo...