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

Phase 4

Completed

• Conditions: Gaucher's Disease
• Interventions: Drug: Cerezyme® / Imiglucerase

• Registration Number: NCT04656600
• Lead Sponsor: Sanofi

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

Detailed Description

Approximatively 14 months including a 12 months treatment period

Study Timeline

• Study First Submitted: 2020-12-04
• Study First Submitted QC: 2020-12-04
• Study First Posted: 2020-12-07
• Study Start: 2021-03-02
• Primary Completion: 2023-10-12
• Study Completion: 2023-10-12
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-21
• Last Update Posted: 2024-01-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 12

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Adverse events	Baseline to the end of 13 months	Number of participants with AEs
Changes in platelet count	Baseline to the end of 12 months	The mean changes in platelet count
Changes in haemoglobin	Baseline to the end of 12 months	The mean changes in haemoglobin

Secondary Outcome Measures

Name	Time	Method
Changes in liver volume	Baseline to the end of 12 months	The mean change of liver volumne
Changes in spleen volume	Baseline to the end of 12 months	The mean change of spleen volumne
Skeletal involvement	Baseline to the end of 12 months	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
Quality of life (QoL)	Baseline to the end of 3 months, 6 months, 9 months and 12 months	The mean change of QoL

Trial Locations

Locations (5)

Investigational Site Number : 102; 🇨🇳 Guangzhou, China

Investigational Site Number : 107; 🇨🇳 Beijing, China

Investigational Site Number : 101; 🇨🇳 Beijing, China

Investigational Site Number : 104; 🇨🇳 Chengdu, China

Investigational Site Number : 105; 🇨🇳 Guangzhou, China