Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ
Status:
Recruiting
Trial end date:
2023-09-01
Target enrollment:
Participant gender:
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