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

A Phase 4, Single-arm, Open-label Safety and Efficacy Study of Aldurazyme® (Laronidase) as Enzyme Replacement Therapy in Participants With Mucopolysaccharidosis I (MPS I) in China

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 Overview

• Status: Completed
• Conditions: Mucopolysaccharidosis I
• Intervention / Treatment: Drug: 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.

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 4

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100730
    • Investigational Site Number :1560002
  • Beijing, China, 100034
    • Investigational Site Number :1560003
  • Guangzhou, China, 510623
    • Investigational Site Number :1560004
  • Hangzhou, China, 310003
    • Investigational Site Number :1560006
  • Wuhan, China, 430030
    • Investigational Site Number :1560001

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Aldurazyme (laronidase); Aldurazyme (laronidase) treatment at approved dose and regimen, administered every week as an IV infusion	Drug: Laronidase; Solution for injection; Intravenous

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Genzyme, a Sanofi Company
• Investigators: Study Director: Clinical Sciences & Operations, Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): October 28, 2021
• Primary Completion (Actual): July 26, 2023
• Study Completion (Actual): July 26, 2023

Study Registration Dates

• First Submitted: October 21, 2021
• First Submitted That Met QC Criteria: November 14, 2021
• First Posted (Actual): November 26, 2021

Study Record Updates

• Last Update Posted (Actual): September 28, 2023
• Last Update Submitted That Met QC Criteria: September 26, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: clinical trial; enzyme replacement therapy; mucopolysaccharidosis I; laronidase
• Additional Relevant MeSH Terms: Metabolic Diseases; Genetic Diseases, Inborn; Connective Tissue Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Mucinoses; Mucopolysaccharidoses; Mucopolysaccharidosis I
• Other Study ID Numbers: LPS16578; U1111-1260-3947 (Registry Identifier: ICTRP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No