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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00635427 | An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease | PHASE3 | COMPLETED | 95 | — | — | Mar 13, 2008 | Dec 28, 2012 | Jun 10, 2021 | 21 | United States, Argentina +9 |
| NCT00430625 | A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease | PHASE3 | COMPLETED | 25 | — | — | Feb 15, 2007 | Apr 1, 2009 | Jun 29, 2021 | 5 | Argentina, Israel +3 |
Safety was evaluated by an analysis of adverse events (AEs), concomitant medication use, clinical laboratory tests, vital signs during the infusion of study drug, physical examination, and the development of anti-velaglucerase alfa. No formal comparisons or statistical tests were applied for the safety analyses, including for differences between the groups.
Efficacy endpoint
| Arm | Type | Description |
|---|---|---|
| VPRIV 60 U/kg(VPRIV Parent Study 45 or 60 U/kg- TKT032,GCB039) | EXPERIMENTAL | This arm is the Overall velaglucerase alfa (VPRIV) 60 U/kg and includes patients from the following groups: VPRIV 45 U/kg or 60 U/kg, IV, EOW for 51 weeks in parent study TKT032 (NCT00430625) and switched to 60 U/kg in HGT-GCB-044 to maintain blindness or 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631) |
| VPRIV 60 U/kg (Parent study-imiglucerase(60 U/kg) HGT-GCB-039) | EXPERIMENTAL | imiglucerase 60 U/kg, IV, EOW for 39 weeks in parent study HGT-GCB-039 (NCT00553631)and switched 60 U/kg VPRIV in HGT-GCB-044 |
| VPRIV 15-60 U/kg (Parent study VPRIV (15-60 U/kg) TKT034) | EXPERIMENTAL | VPRIV 15- 60 U/kg, IV, EOW for 51 weeks in parent study TKT034 (NCT00478647) and continued in HGT-GCB-044 at the same dose as prescribed in TKT034 |
| VPRIV®-45 U/kg, IV, every other week | EXPERIMENTAL | VPRIV® (velaglucerase alfa, Gene Activated® human glucocerebrosidase, GA-GCB) |
| VPRIV®-60 U/kg, IV, every other week | EXPERIMENTAL | VPRIV® (velaglucerase alfa, Gene Activated® human glucocerebrosidase,GA-GCB) |
| Name | Type | Description |
|---|---|---|
| VPRIV® | BIOLOGICAL | Intravenous infusion, every other week (EOW) |
| VPRIV ®, | BIOLOGICAL | Intravenous (IV) infusion, every other week via intravenous infusion for 12 months |
Inclusion Criteria: 1. The patient has completed study TKT032 or TKT034, or study HGT-GCB-039. 2. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at...