NCT05134571

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

A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in China

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: 5 Years
• Healthy volunteers: Not accepted

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).

Detailed description

Study duration for each participant will be a total of 29 weeks which will include 2 weeks of screening, 26 weeks of treatment period and 1 week of follow-up period.

Conditions

• Mucopolysaccharidosis I

Interventions

Type	Name	Description
DRUG	Laronidase	Solution for injection; Intravenous

Timeline

• Start date: 2021-10-28
• Primary completion: 2023-07-26
• Completion: 2023-07-26
• First posted: 2021-11-26
• Last updated: 2025-09-17

Locations

5 sites across 1 country: China