Phase 3 Mucopolysaccharidosis I 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
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
Mucopolysaccharidosis II
Denali Therapeutics Inc.63 enrolled32 locationsNCT05371613