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

NCT05054387 | Completed Phase 4

• 2 other identifiers: LPS16583U1111-1255-4881
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 6

Brief Summary

This is a 54-week Phase 4, open label, single arm study to evaluate the safety and the efficacy of Fabrazyme (agalsidase beta) as enzyme replacement therapy (ERT) in Chinese participants with Fabry Disease.

Conditions

Fabry Disease

Outcome Measures

Primary Outcomes (1)

• Incidence of treatment-emergent adverse events (AEs)

  Including TEAE, SAEs, and adverse events of special interest (AESIs) including infusion associated reactions (IARs) and change of clinical laboratory, vital signs and ECG

  Baseline to week 50

Secondary Outcomes (9)

• The absolute changes of plasma globotriaosylsphingosine (lyso-GL3)

  from baseline to Week 6, Week 12, Week 24 and Week 48

• The percent changes of plasma lyso-GL3

  from baseline to Week 6, Week 12, Week 24 and Week 48

• The absolute changes of plasma globotriaosylceramide (GL3)

  from baseline to Week 6, Week 12, Week 24 and Week 48

• The percent changes of plasma GL3

  from baseline to Week 6, Week 12, Week 24 and Week 48

• The number of participants with abnormal plasma GL3 values per central lab reference range

  at Week 6, Week 12, Week 24 and Week 48

Study Arms (1)

Agalsidase beta

EXPERIMENTAL

Agalsidase beta treatment at approved dose and regimen, administered once every 2 weeks as an IV infusion

Drug: Agalsidase beta

Interventions

Agalsidase beta DRUG

Powder for concentration into a solution Intravenous (IV) infusion

Also known as: GZ419828 Fabrazyme

Agalsidase beta

Eligibility Criteria

• Age: 8 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participant must be 8 years of age or older, at the time of signing the informed consent
• Participants naive to agalsidase beta and agalsidase alpha
• Chinese participants diagnosed with Fabry disease and with documented plasma or leukocyte αGAL activity deficient below laboratory's reference range, and/or documented diagnosis by genotyping
• Participants must have one or more symptoms and signs consistent with manifestations of Fabry disease (not limited to neuropathic pain, chronic kidney disease, hypertrophic cardiomyopathy, cardiac rhythm disturbances, cerebrovascular involvement, cornea verticillata, angiokeratoma, gastrointestinal symptoms, hypo- or anhydrosis)
• A female participant is eligible to participate if she is not pregnant or breastfeeding and use an acceptable contraceptive method
• Participants and/or participant's legal representative capable of giving signed informed consent.

You may not qualify if:

• The participant has undergone kidney transplantation.
• The participant has a clinically significant organic disease (with the exception of symptoms relating to Fabry disease) in the opinion of the Investigator, would preclude participation in the trial.
• Received an investigational drug, or device, other than Fabrazyme, within 30 days of anticipated IMPs administration or 5 half-lives of the previous investigational drug, whichever is longer.
• The patient has current evidence of kidney failure or renal insufficiency, as defined by eGFR \<30 mL/min/1.73 m2.
• Individuals who have life threatening hypersensitivity (anaphylactic reaction) to the active substance or any of the excipients included.
• The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Sponsors & Collaborators

• Genzyme, a Sanofi Company: lead

Study Sites (6)

Investigational Site Number :1560003

Beijing, 100034, China

Location

Investigational Site Number :1560002

Beijing, 100730, China

Location

Investigational Site Number :1560001

Shanghai, 200025, China

Location

Investigational Site Number :1560004

Shanghai, 201102, China

Location

Investigational Site Number :1560006

Taiyuan, 030001, China

Location

Investigational Site Number :1560005

Wuhan, 016040, China

Location

Related Publications (1)

• Ren H, Zhang W, Ouyang Y, Guo J, Xu H, Ma J, Luo X, Pan X, Yuan Y, Zhang W, Shen Q, Li B, Feng Q, Liu S, Chen N. A phase 4, open-label, multicenter study of the safety and efficacy of agalsidase beta in Chinese patients with Fabry disease. Orphanet J Rare Dis. 2025 Aug 4;20(1):401. doi: 10.1186/s13023-025-03950-7.

  PMID: 40760696 DERIVED

Related Links

• LPS16583 Plain Language Results Summary

MeSH Terms

Conditions

Fabry Disease

Interventions

agalsidase beta

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolism, Inborn Errors; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

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: September 9, 2021
• First Posted: September 23, 2021
• Study Start: October 13, 2021
• Primary Completion: March 9, 2023
• Study Completion: March 9, 2023
• Last Updated: September 11, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

CN (6)