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

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

Timeline

Start

2017-03-30

Primary completion

2017-10-04

Completion

2017-10-04

Publications

• Okuyama T, Eto Y, Sakai N, Minami K, Yamamoto T, Sonoda H, Yamaoka M, Tachibana K, Hirato T, Sato Y. Iduronate-2-Sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. Mol Ther. 2019 Feb 6;27(2):456-464. doi: 10.1016/j.ymthe.2018.12.005. Epub 2018 Dec 8.

Drugs

Indications