Gaucher disease (GD)
Conditions
Interventions
Observation group:none
Sponsors
Beijing Childrens Hospital,Capital Medical University
Eligibility
Sex/Gender
All
Age
4 Years to No maximum
Inclusion criteria
Inclusion criteria: 1.Has a documented, confirmed diagnosis of type 1 Gaucher disease: decreased GCB activity level that is 30% of normal, but with confirmation of genetic mutation test; 2.Patients have been treated with velaglucerase alfa for >=9 months consecutively, and in the past 9 months, velaglucerase alfa has been the only Gaucher disease-specific treatment used; 3.>=4 years old;
Exclusion criteria
Exclusion criteria: 1.The patient or his/her guardian did not sign the informed consent form; 2.Patients who have previously participated in the velaglucerase alfa PAC study (TAK-669-3001); patients who have used other Gaucher disease-specific therapies in the last 9 months; and patients who have participated in gene therapy clinical trials.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Hemoglobin;platelet; | — |
Secondary
| Measure | Time frame |
|---|---|
| Liver volume;bone density;height;Bone pain;Lyso-Gb1;Spleen volume;Quality of life score;Adverse events; | — |
Countries
China
Contacts
Public ContactYanlong Duan
Beijing Childrens Hospital,Capital Medical University
Outcome results
None listed