Phase 2 Mucopolysaccharidosis Type 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
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