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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02782741 | Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease | PHASE3 | COMPLETED | 101 | — | — | Nov 2, 2016 | May 31, 2023 | Apr 4, 2024 | 69 | United States, Argentina +23 |
FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC is the volume of air (in liters) that can be forcibly blown out after full inspiration in the upright position. Least square (LS) mean and standard error (SE) were derived from mixed model for repeated measure (MMRM) model with baseline FVC \[percent (%) predicted, as continuous\], sex, age (in years at baseline), treatment group, visit, interaction term between treatment group and visit as fixed effects. Percent of predicted FVC = (actual FVC measurement)/(predicted value of FVC) \* 100. After non-inferiority (NI) testing, a test for superiority of avalglucosidase alfa versus alglucosidase alfa was performed with an overall 2-sided 5% level of significance.
| Arm | Type | Description |
|---|---|---|
| avalglucosidase alfa (GZ402666) | EXPERIMENTAL | Administered intravenously every 2 weeks |
| alglucosidase alfa (GZ419829) | ACTIVE_COMPARATOR | Administered intravenously every 2 weeks |
| Name | Type | Description |
|---|---|---|
| Avalglucosidase alfa (GZ402666) | DRUG | Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous |
| Alglucosidase alfa (GZ419829) | DRUG | Pharmaceutical form: powder for concentrate for solution for infusion Route of administration: intravenous |
Inclusion criteria : * The participant has confirmed acid alpha-glucosidase (GAA) enzyme deficiency from any tissue source and/or 2 confirmed GAA gene mutations. * The participant must provide signed, informed consent prior to performing any study related procedures. Consent of a legally authorized...