China Post-Marketing Surveillance (PMS) Study of Aldurazyme®
study id #: NCT05134571
condition: Mucopolysaccharidosis I
status: Recruiting
purpose: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).
intervention: Laronidase
results: https://clinicaltrials.gov/ct2/show/results/NCT05134571
last updated: March 05, 2022
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