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

NCT05134571 | Completed Phase 4

• 2 other identifiers: LPS16578U1111-1260-3947
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 5

Brief 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).

Conditions

Mucopolysaccharidosis I

Keywords

clinical trial; enzyme replacement therapy; mucopolysaccharidosis I; laronidase

Outcome Measures

Primary Outcomes (3)

• 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

• 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

• The percent change of uGAGs

  Baseline to Week 26

Secondary Outcomes (3)

• The percent change of uGAGs

  Baseline to Week 2, Week 4, Week 8, Week 12 and Week 20

• The absolute change of uGAGs

  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)

  Baseline to Week 26

Study Arms (1)

Aldurazyme (laronidase)

EXPERIMENTAL

Aldurazyme (laronidase) treatment at approved dose and regimen, administered every week as an IV infusion

Drug: Laronidase

Interventions

Laronidase DRUG

Solution for injection; Intravenous

Aldurazyme (laronidase)

Eligibility Criteria

• Age: 5 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Chinese participants have a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and fibroblast or leukocyte IDUA (iduronidase) activity \<10% of normal.
• Participants have to be able to stand independently and walk a minimum of 5 meters in 6 minutes.
• A female participant is eligible to participate if she is not pregnant or breastfeeding, and at least one of the following conditions applies:
• Is not a woman of childbearing potential (WOCBP).
• Is a WOCBP and agrees to use an acceptable contraceptive method during the intervention period and at a minimum until 7 days after the last dose of study intervention.
• A WOCBP must have a negative highly sensitive pregnancy test (urine or serum as required by local regulations) within the screening period before the first dose of study intervention.
• If a urine test cannot be confirmed as negative (eg, an ambiguous result), a serum pregnancy test is required. In such cases, the participant must be excluded from participation if the serum pregnancy result is positive.
• Contraceptive/barrier method is not applicable for male participants.

You may not qualify if:

• Prior tracheostomy or bone marrow transplantation or hematopoietic stem cell transplantation.
• Have a plan to undergo bone marrow transplantation or hematopoietic stem cell transplantation within half a year after enrollment.
• Received an investigational drug, or device, other than Aldurazyme, within 30 days prior to study enrollment.
• Received an investigational gene therapy.
• The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (5)

Investigational Site Number :1560003

Beijing, 100034, China

Location

Investigational Site Number :1560002

Beijing, 100730, China

Location

Investigational Site Number :1560004

Guangzhou, 510623, China

Location

Investigational Site Number :1560006

Hangzhou, 310003, China

Location

Investigational Site Number :1560001

Wuhan, 430030, China

Location

Related Publications (1)

• Liang Y, Yang YL, Zou CC, Liu L, Jiao Y, Wang Y, Liu X, Luo XP. Safety and efficacy of laronidase in Chinese patients with mucopolysaccharidosis type I: a phase IV, single-arm, open-label, multicenter study. Orphanet J Rare Dis. 2025 Oct 29;20(1):547. doi: 10.1186/s13023-025-04056-w.

  PMID: 41163043 DERIVED

Related Links

• LPS16578 Plain Language Results Summary

MeSH Terms

Conditions

Mucopolysaccharidosis I

Interventions

Iduronidase

Condition Hierarchy (Ancestors)

Mucopolysaccharidoses; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lysosomal Storage Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Glycoside Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes

Study Officials

• Clinical Sciences & Operations

  Sanofi

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 21, 2021
• First Posted: November 26, 2021
• Study Start: October 28, 2021
• Primary Completion: July 26, 2023
• Study Completion: July 26, 2023
• Last Updated: September 17, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

CN (5)