Phase 2 Sanfilippo Syndrome Clinical Trials
3 recruitingPhase 2
What is a Phase 2 trial?
Phase 2 trials evaluate whether a treatment works for a specific condition and further assess its safety. These studies typically enroll several hundred participants and provide preliminary data on effectiveness.
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