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| Name | Class |
|---|---|
| Sanofi | INDUSTRY |
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Evaluation of the safety in the combination usage of Cerdelga and Cerezyme in type III Gaucher disease patients and the efficacy on soft tissue diseases.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental Case_Eiglustat | Experimental | Besides regular ERT, patients also need to take Eiglustat for 24 months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eliglustat | Drug |
|
| Measure | Description | Time Frame |
|---|---|---|
| Adverse Events | Number of adverse events in patients. | From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first. |
| Measure | Description | Time Frame |
|---|---|---|
| Assessment of Gaucher related biomarkers test :CCL18 (30% decrease) | Measure of Gaucher disease type I biomarkers:CCL18(ng/ml) in plasma. | Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga. |
| Assessment of Gaucher related biomarkers test :Lyso GL1(30% decrease) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Ni-Chung Lee, M.D., Ph.D | National Taiwan University Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Taiwan University Hospital | Taipei | 10041 | Taiwan |
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| ID | Term |
|---|---|
| D005776 | Gaucher Disease |
| ID | Term |
|---|---|
| D013106 | Sphingolipidoses |
| D020140 | Lysosomal Storage Diseases, Nervous System |
| D020739 | Brain Diseases, Metabolic, Inborn |
| D001928 | Brain Diseases, Metabolic |
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| ID | Term |
|---|---|
| C522917 | eliglustat |
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Measure of Gaucher disease type I biomarkers:Lyso GL1 (ng/mL) in plasma |
| Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga. |
| Assessment of Gaucher related biomarkers test:Chitotriosidase(30% decrease) | Measure of Gaucher disease type I biomarkers:Chitotriosidase(nmol/ml/h) in plasma. | Baseline,1,3,6,12,18 and 24 months after receiving Cerdelga. |
| Change in lymphadenopathy manifestations. | Physician will pay close attention to the lymphadenopathy including size, location, and number of enlarged lymph nodes, evaluate by palpation and radiological (MRI examination). The total size of the lympadenopathy will be combined into one report as "Total size" cm^3. | From date of enrollment with information consent form until 24 months or date of death from any cause, whichever came first. |
| Pharmacokinetics | Eliglustat plasma concentration over time | Plasma concentration-time data will be obtained pre-dose (within 30 minutes prior to dosing) and at 1, 2, 6, 12, 26, 36 hours after 1st dosing, and 1,3,6,12,18 and 24 months thereafter. |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D008661 | Metabolism, Inborn Errors |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D008064 | Lipidoses |
| D008052 | Lipid Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D052439 | Lipid Metabolism Disorders |