China Post-marketing Surveillance (PMS) Study of Aldurazyme®
Status:
Recruiting
Trial end date:
2023-08-24
Target enrollment:
Participant gender:
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).