Phase 2 Mucopolysaccharidosis III 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 1Phase 2
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis III-B
JCR Pharmaceuticals Co., Ltd.10 enrolled3 locationsNCT06488924