Clinical Trial: A Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional



Official Title: A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Brief Summary:

The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below,

  • to collect the safety information of JR-141
  • to evaluate the plasma pharmacokinetics of JR-141
  • to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms