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

A Single Arm, Prospective, Open Label, Multicenter Study to Evaluate Efficacy and Safety of One-year Maximum Dosage in Chinese Label of Imiglucerase Treatment in Chinese Patients Who Are Diagnosed as Gaucher Disease Type Ⅲ

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

Study Overview

• Status: Completed
• Conditions: Gaucher's Disease
• Intervention / Treatment: Drug: Cerezyme® / Imiglucerase

Detailed Description

Approximatively 14 months including a 12 months treatment period

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

Contacts and Locations

Study Locations

• China
  • Beijing, China, 100020
    • Investigational Site Number : 107
  • Beijing, China, 100032
    • Investigational Site Number : 101
  • Chengdu, China, 610041
    • Investigational Site Number : 104
  • Guangzhou, China, 510080
    • Investigational Site Number : 102
  • Guangzhou, China, 510623
    • Investigational Site Number : 105

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion criteria:

• 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.

Exclusion criteria:

• 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.

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: Cerezyme® / Imiglucerase; Cerezyme® (imiglucerase for injection) is administered by intravenous infusion, 60 U/kg once every 2 weeks.	Drug: Cerezyme® / Imiglucerase; Pharmaceutical form: lyophilisate for solution for infusion Route of administration: intravenous

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in spleen volume	The mean change of spleen volumne	Baseline to the end of 12 months
Changes in liver volume	The mean change of liver volumne	Baseline to the end of 12 months
Skeletal involvement	The changes in frequency, duration and severity of bone pain and number of bone crisis The changes in frequency, duration and severity of bone pain and number of bone crisisThe changes in frequency, duration and severity of bone pain and number of bone crisis	Baseline to the end of 12 months
Quality of life (QoL)	The mean change of QoL	Baseline to the end of 3 months, 6 months, 9 months and 12 months

Collaborators and Investigators

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

Publications and helpful links

Helpful Links

• LPS16031 Plain Language Results Summary

Study record dates

Study Major Dates

• Study Start (Actual): March 2, 2021
• Primary Completion (Actual): October 12, 2023
• Study Completion (Actual): October 12, 2023

Study Registration Dates

• First Submitted: December 4, 2020
• First Submitted That Met QC Criteria: December 4, 2020
• First Posted (Actual): December 7, 2020

Study Record Updates

• Last Update Posted (Estimated): September 15, 2025
• Last Update Submitted That Met QC Criteria: September 9, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Lipid Metabolism Disorders; Lysosomal Storage Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases, Nervous System; Sphingolipidoses; Lipidoses; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Gaucher Disease; imiglucerase
• Other Study ID Numbers: LPS16031; U1111-1244-1166 (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