Primary Objective:
Evaluate the efficacy and safety of eliglustat in Chinese pediatric patients (≥12 to \<18 years old) with Gaucher disease type 1 and type 3.
Secondary Objective:
Evaluate the quality of life in Chinese pediatric patients (≥12 to \<18 years old) with Gaucher disease type 1 and type 3 treated with eliglustat.
The patient is ≥12 to <18 years old at the time of informed consent.
Glucocerebrosidase (GBA) activity reduced to ≤30% of the lower limit of normal, or
GBA activity reduced by >30% of the lower limit of normal, but confirmed by glucocerebrosidase (GBA) genotype.
Postmenarchal female patients must have a documented negative pregnancy test prior to enrollment and throughout the study.
Underwent substrate reduction therapy (SRT) for GD or received miglustat treatment within 12 months prior to enrollment.
Underwent partial or total splenectomy prior to enrollment or experienced active, clinically significant splenic infarction within the previous 12 months.
The patient is transfusion-dependent; has a history of esophageal varices or liver infarction; elevated liver enzymes; significant congenital cardiac defect; coronary artery disease; left-sided heart failure; clinically significant arrhythmias; or conduction defects such as Type 2 second-degree or third-degree atrioventricular (AV) block, complete bundle branch block, prolonged QTc interval, or sustained ventricular tachycardia (VT).
Presence of significant comorbidities, as determined by the Principal Investigator, which may affect study data or confound study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver disease, pulmonary complications, cardiac structural or functional abnormalities, etc.).
Single group
Eliglustat Tartrate Capsules
DrugThe initial dose is 42 mg taken orally twice a day. After 2 weeks of treatment, if the blood trough concentration is less than 5 ng/mL, the dose will be increased to 84 mg taken orally twice daily.
Changes in hemoglobin level
Absolute change from baseline for hemoglobin (g/dL)
Changes in platelet count
Percent change from baseline for platelet count
Changes in spleen volume
Percent change from baseline for spleen volume
Changes in liver volume
Percent change from baseline for liver volume
Changes in Quality of Life
Health-related quality of life will be measured by the Pediatric Quality of Life Inventory™ (PedsQL™) questionnaires
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