NCT04656600

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

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

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Sanofi · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

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

Conditions

• Gaucher's Disease

Interventions

Type	Name	Description
DRUG	Cerezyme® / Imiglucerase	Pharmaceutical form: lyophilisate for solution for infusion Route of administration: intravenous

Timeline

• Start date: 2021-03-02
• Primary completion: 2023-10-12
• Completion: 2023-10-12
• First posted: 2020-12-07
• Last updated: 2025-09-15

Locations

5 sites across 1 country: China