China Post-marketing Surveillance (PMS) Study of Aldurazyme®

Summary

This is a single treatment arm study that is open-label to be conducted in Chinese participants with MPS I. Trial Objectives are to evaluate the safety and tolerability of Aldurazyme in Chinese MPS I participants, and to evaluate the efficacy of Aldurazyme on the percent change of urinary glycosaminoglycans (uGAGs) from baseline to Week 26. The study will also evaluate the effect on uGAG level and liver volume (hepatomegaly) after 26 weeks, with Aldurazyme treatment in Chinese MPS I participants. Treatment duration will include: 2 weeks of screening, 26 weeks of treatment and 1 week of follow-up period. During the treatment period, weekly visits are designed to accommodate weekly administration of Aldurazyme (laronidase).

Timeline

Start

2021-10-28

Primary completion

2023-07-26

Completion

2023-07-26

Publications

• Liang Y, Yang YL, Zou CC, Liu L, Jiao Y, Wang Y, Liu X, Luo XP. Safety and efficacy of laronidase in Chinese patients with mucopolysaccharidosis type I: a phase IV, single-arm, open-label, multicenter study. Orphanet J Rare Dis. 2025 Oct 29;20(1):547. doi: 10.1186/s13023-025-04056-w.

Drugs

Indications