Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00478647 | Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase | PHASE2 | COMPLETED | 40 | — | — | Jul 25, 2007 | Jun 26, 2009 | Jun 10, 2021 | 15 | United States, Israel +3 |
| NCT00391625 | Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT) | PHASE1 | COMPLETED | 10 | — | — | Sep 13, 2004 | Jan 31, 2008 | Jun 22, 2021 | 3 | Israel, Romania +1 |
Safety was assessed throughout the study by assessments including adverse events, concomitant medication use, and vital signs. Additional safety assessments, including 12-lead ECGs, physical examinations, clinical laboratory tests and determination of the presence of anti-velaglucerase alfa antibodies. Refer to Adverse event section for further details.
Overall Summary of Treatment-emergent Adverse Events-Safety Population
| Arm | Type | Description |
|---|---|---|
| GA-GCB (velaglucerase alfa) | EXPERIMENTAL | 15-60 U/kg, every other week via intravenous infusion |
| GA-GCB | EXPERIMENTAL | 15-60 U/kg every other week via intravenous infusion |
| Name | Type | Description |
|---|---|---|
| GA-GCB (velaglucerase alfa) | BIOLOGICAL | 15-60 U/kg, every other week via intravenous infusion |
| GA-GCB | DRUG | 15-60 U/kg every other week via intravenous infusion |
Inclusion Criteria: Includes: * The participant has a documented diagnosis of type 1 Gaucher disease, as determined by deficient glucocerebrosidase (GCB) activity relative to normal as measured in leukocytes or by genotype analysis and the participant/legal guardian is willing and able to provide ...