Phase 2 MPS I Clinical Trials
2 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 1–2 of 2 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 2
Pentosan Polysulfate Sodium (PPS) in subjects with mucopolysaccharidosis type I (MPS I)
Mucopolysaccharidosis type I (MPS I)
Paradigm Biopharmaceuticals Pty Ltd10 enrolled1 locationACTRN12620000823976