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).
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Solution for injection; Intravenous
Investigational Site Number :1560003
Beijing, China
Investigational Site Number :1560002
Beijing, China
Investigational Site Number :1560004
Guangzhou, China
Investigational Site Number :1560006
Hangzhou, China
Investigational Site Number :1560001
Wuhan, China
Participants with adverse events (AEs)
Incidence of AEs, serious adverse events (SAEs) and adverse events of special interest (AESIs) including infusion associated reactions (IARs) during the treatment emergent (TE) period
Time frame: Baseline to Week 27
The incidence of Potentially clinically significant abnormality (PCSA) analyses for clinical laboratory, vital signs, and ECG parameters during the TE period
Time frame: Baseline to Week 27
The percent change of uGAGs
Time frame: Baseline to Week 26
The percent change of uGAGs
Time frame: Baseline to Week 2, Week 4, Week 8, Week 12 and Week 20
The absolute change of uGAGs
Time frame: Baseline to Week 2, Week 4, Week 8, Week 12, Week 20 and Week 26
The percent change of liver volume (Abdominal B type ultrasound examination)
Time frame: Baseline to Week 26
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