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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT05134571
Other study ID # LPS16578
Secondary ID U1111-1260-3947
Status Completed
Phase Phase 4
First received
Last updated
Start date October 28, 2021
Est. completion date July 26, 2023

Study information

Verified date September 2023
Source Sanofi
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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).


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.


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Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Laronidase
Solution for injection; Intravenous

Locations

Country Name City State
China Investigational Site Number :1560002 Beijing
China Investigational Site Number :1560003 Beijing
China Investigational Site Number :1560004 Guangzhou
China Investigational Site Number :1560006 Hangzhou
China Investigational Site Number :1560001 Wuhan

Sponsors (1)

Lead Sponsor Collaborator
Genzyme, a Sanofi Company

Country where clinical trial is conducted

China, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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 Baseline to Week 27
Primary The incidence of Potentially clinically significant abnormality (PCSA) analyses for clinical laboratory, vital signs, and ECG parameters during the TE period Baseline to Week 27
Primary The percent change of uGAGs Baseline to Week 26
Secondary The percent change of uGAGs Baseline to Week 2, Week 4, Week 8, Week 12 and Week 20
Secondary The absolute change of uGAGs Baseline to Week 2, Week 4, Week 8, Week 12, Week 20 and Week 26
Secondary The percent change of liver volume (Abdominal B type ultrasound examination) Baseline to Week 26
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