Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ

NCT04656600 | Completed Phase 4

• 2 other identifiers: LPS16031U1111-1244-1166
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 5

Brief Summary

Primary Objective

• To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
• To evaluate the safety profile of imiglucerase in maximum dose in the label (60U/kg, IV biweekly) in Chinese patients. Secondary Objective
• To evaluate the efficacy on viscera manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
• To evaluate the efficacy on bone disease of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ
• To evaluate the effect on quality of life of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type Ⅲ

Conditions

Gaucher's Disease

Outcome Measures

Primary Outcomes (3)

• Changes in haemoglobin

  The mean changes in haemoglobin

  Baseline to the end of 12 months

• Changes in platelet count

  The mean changes in platelet count

  Baseline to the end of 12 months

• Adverse events

  Number of participants with AEs

  Baseline to the end of 13 months

Secondary Outcomes (4)

• Changes in spleen volume

  Baseline to the end of 12 months

• Changes in liver volume

  Baseline to the end of 12 months

• Skeletal involvement

  Baseline to the end of 12 months

• Quality of life (QoL)

  Baseline to the end of 3 months, 6 months, 9 months and 12 months

Study Arms (1)

Cerezyme® / Imiglucerase

EXPERIMENTAL

Cerezyme® (imiglucerase for injection) is administered by intravenous infusion, 60 U/kg once every 2 weeks.

Drug: Cerezyme® / Imiglucerase

Interventions

Cerezyme® / Imiglucerase DRUG

Pharmaceutical form: lyophilisate for solution for infusion Route of administration: intravenous

Cerezyme® / Imiglucerase

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Capable of giving signed informed consent.
• Participant is diagnosed with GD type Ⅲ
• Participant with neurological manifestations
• Participant whose age is \> 2 years old.
• Participant whose spleen and/or liver volume is \> ULN at Screening.

You may not qualify if:

• Major congenital anomaly
• Clinically significant intercurrent organic disease unrelated to Gaucher disease, which means the disease or condition that may have impact on the parameters chosen for primary endpoints (e.g. level of hemoglobin platelets, liver/spleen enlargement and bone pains)
• Prior treatment with ERT.
• Physical conditions that cannot tolerate regular treatment or follow-up visit.
• Pregnant or lactating women
• Participant is participating in or has participated in another clinical study using any investigational therapy in 3 months
• Participant has been diagnosed with central nervous system disease unrelated to Gaucher disease, or MRI result of the participant indicates space-occupying lesion in central nervous system
• The patient has a documented hemoglobinopathies, deficiency of iron, vitamin B-12, or folate that requires treatment not yet initiated or, if initiated, the patient has not been stable under treatment for at least 6 months prior to administration of the first dose of Cerezyme in this study
• Participant not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or participants potentially at risk of noncompliance to study procedures
• Any specific situation during study implementation/course that may rise ethics considerations
• Sensitivity to any of the study interventions, or components thereof, or drug or other allergy that, in the opinion of the Investigator, contraindicates participation in the study
• The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (5)

Investigational Site Number : 107

Beijing, 100020, China

Location

Investigational Site Number : 101

Beijing, 100032, China

Location

Investigational Site Number : 104

Chengdu, 610041, China

Location

Investigational Site Number : 102

Guangzhou, 510080, China

Location

Investigational Site Number : 105

Guangzhou, 510623, China

Location

Related Links

• LPS16031 Plain Language Results Summary

MeSH Terms

Conditions

Gaucher Disease

Interventions

imiglucerase

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; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; 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: December 4, 2020
• First Posted: December 7, 2020
• Study Start: March 2, 2021
• Primary Completion: October 12, 2023
• Study Completion: October 12, 2023
• Last Updated: September 15, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will share

Locations

CN (5)