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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01411228 | A Multicenter Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease | PHASE3 | COMPLETED | 15 | — | — | Sep 1, 2011 | Aug 1, 2014 | Sep 7, 2018 | 3 | Israel, Paraguay +1 |
| NCT01422187 | A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease | PHASE3 | COMPLETED | 19 | — | — | Aug 1, 2011 | Sep 1, 2014 | Apr 19, 2023 | - | — |
| NCT00712348 | Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase | PHASE3 | COMPLETED | 31 | — | — | Dec 1, 2008 | May 1, 2013 | Oct 4, 2018 | 10 | United States, Australia +4 |
| NCT00705939 | Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial | PHASE3 | COMPLETED | 45 | — | — | Jun 1, 2008 | Aug 1, 2013 | Oct 4, 2018 | 11 | United States, Australia +6 |
Median and interquartile range. Baseline is the value obtained from the parent study: PB-06-005 for 30 and 60 Units/kg arms and PB-06-002 from Switchover arm.
Spleen volume measured by MRI
| Arm | Type | Description |
|---|---|---|
| 60 Units/kg | EXPERIMENTAL | - |
| 30 Units/kg | EXPERIMENTAL | - |
| Taliglucerase alfa 30 units/kg | EXPERIMENTAL | Subjects randomized to receive 30 units/kg |
| Taliglucerase alfa 60 units/kg | EXPERIMENTAL | Subjects randomized to 60 units/kg |
| Taliglucerase alfa | EXPERIMENTAL | Open label taliglucerase alfa treatment |
| Naive 30 Units/kg | EXPERIMENTAL | Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) |
| Naive 60 Units/kg | EXPERIMENTAL | Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) |
| Switchover | EXPERIMENTAL | Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348) |
| Name | Type | Description |
|---|---|---|
| Taliglucerase alfa | DRUG | Taliglucerase alfa for infusion every two weeks for 24 months |
Inclusion Criteria: * Successful completion of Protocol PB-06-002 or PB-06-005 * The subject, parent(s) or legal guardian(s) signs an informed consent and/or assent Exclusion Criteria: * Currently taking another investigational drug for any condition. * Presence of neurological signs and symptoms...