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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01898364 | Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients. | PHASE1 | COMPLETED | 24 | — | — | Aug 19, 2013 | Feb 25, 2015 | Mar 23, 2023 | 17 | United States, Belgium +5 |
| Arm | Type | Description |
|---|---|---|
| GZ402666 (neoGAA) Group 1 - 5 mg | EXPERIMENTAL | Intravenous infusion of 5mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks |
| GZ402666 (neoGAA) Group 1 - 10 mg | EXPERIMENTAL | Intravenous infusion of 10mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks. |
| GZ402666 (neoGAA) Group 1 - 20 mg | EXPERIMENTAL | Intravenous infusion of 20mg neoGAA to treatment naïve late onset Pompe disease patients once every other week for a total of 24 weeks. |
| GZ402666 (neoGAA) Group 2 - 5 mg | EXPERIMENTAL | Intravenous infusion of 5mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa. |
| GZ402666 (neoGAA) Group 2 - 10 mg | EXPERIMENTAL | Intravenous infusion of 10mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa. |
| GZ402666 (neoGAA) Group 2 - 20 mg | EXPERIMENTAL | Intravenous infusion of 20mg neoGAA once every other week for a total of 24 weeks to late onset Pompe disease patients previously treated with alglucoside alfa. |
| Name | Type | Description |
|---|---|---|
| GZ402666 | DRUG | Pharmaceutical form:lyophilized powder reconstituted for infusion Route of administration: intravenous |
Inclusion criteria : For both Group 1 and Group 2: * Male or female patients with confirmed acid α-glucosidase (GAA) enzyme deficiency from any tissue source and/or confirmed GAA gene mutation and without known cardiac hypertrophy. * Patient willing and able to provide signed informed consent * Pa...