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

NCT ID: NCT05134571

Last Updated: 2025-09-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-10-28
• Study Completion Date: 2023-07-26

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).

Detailed 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.

Conditions

Mucopolysaccharidosis I

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Aldurazyme (laronidase)

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

Group Type: EXPERIMENTAL

Laronidase

Intervention Type: DRUG

Solution for injection; Intravenous

Interventions

Laronidase

Solution for injection; Intravenous

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

• 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).
• OR
• 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.

Exclusion Criteria

• 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.

• Minimum Eligible Age: 5 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Sciences & Operations

Role: STUDY_DIRECTOR

Sanofi

Locations

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

Countries

China

References

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.

Reference Type: DERIVED

PMID: 41163043 (View on PubMed)

Related Links

https://www.trialsummaries.com/Study/StudyDetails?id=25282&tenant=MT_SNY_9011

LPS16578 Plain Language Results Summary

Other Identifiers

U1111-1260-3947

Identifier Type: REGISTRY

Identifier Source: secondary_id

LPS16578

Identifier Type: -

Identifier Source: org_study_id