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
Approximatively 14 months including a 12 months treatment period
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
Pharmaceutical form: lyophilisate for solution for infusion Route of administration: intravenous
Investigational Site Number : 107
Beijing, China
Investigational Site Number : 101
Beijing, China
Investigational Site Number : 104
Chengdu, China
Investigational Site Number : 102
Guangzhou, China
Investigational Site Number : 105
Guangzhou, China
Changes in haemoglobin
The mean changes in haemoglobin
Time frame: Baseline to the end of 12 months
Changes in platelet count
The mean changes in platelet count
Time frame: Baseline to the end of 12 months
Adverse events
Number of participants with AEs
Time frame: Baseline to the end of 13 months
Changes in spleen volume
The mean change of spleen volumne
Time frame: Baseline to the end of 12 months
Changes in liver volume
The mean change of liver volumne
Time frame: 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
Time frame: Baseline to the end of 12 months
Quality of life (QoL)
The mean change of QoL
Time frame: Baseline to the end of 3 months, 6 months, 9 months and 12 months
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