Phase 2 mucopolysaccharidosis Clinical Trials
6 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 1–6 of 6 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
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
Recruiting
Phase 2Phase 3
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Metabolic DiseasesMucopolysaccharidosis Type II
AO GENERIUM32 enrolled5 locationsNCT05208281
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