Phase 3 Sanfilippo Syndrome Clinical Trials
3 recruitingPhase 3
What is a Phase 3 trial?
Phase 3 trials compare the new treatment against the current standard of care in large groups, often hundreds to thousands of participants. Successful Phase 3 results are typically required for regulatory approval.
Showing 1–3 of 3 trials
Recruiting
Phase 2Phase 3
Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
Mucopolysaccharidosis IIIMPS IIIASanfilippo Syndrome+1 more
Ultragenyx Pharmaceutical Inc36 enrolled5 locationsNCT02716246
Recruiting
Phase 2Phase 3
Study of Cannabidiol in Sanfilippo Syndrome
Mucopolysaccharidosis IIISanfilippo Syndrome
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center35 enrolled1 locationNCT06333041
Recruiting
Phase 2Phase 3
An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III
Sanfilippo SyndromeMPS3
Ozlem Goker-Alpan10 enrolled1 locationNCT06614894