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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03911505 | ZIP Study-OL Study of Safety, PK, Efficacy, PD, Immunogenicity of ATB200/AT2221 in Pediatrics Aged 0 to < 18 y.o. w/LOPD | PHASE3 | ACTIVE NOT_RECRUITING | 21 | — | — | Feb 13, 2020 | Jun 1, 2026 | Oct 27, 2025 | 17 | United States, Australia +4 |
| NCT03729362 | A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease | PHASE3 | COMPLETED | 125 | — | — | Dec 4, 2018 | Jan 15, 2021 | Sep 11, 2025 | 73 | United States, Argentina +22 |
The efficacy of cipaglucosidase alfa/miglustat co-administration on ambulatory function was measured by the 6MWT. The 6MWD, measured in meters, is the distance walked on the 6MWT. A greater distance indicated greater endurance. An increase from baseline indicated improvement.
| Arm | Type | Description |
|---|---|---|
| Cipaglucosidase Alfa (ATB200)/Miglustat(AT2221) | EXPERIMENTAL | Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat (AT2221) capsule |
| Cipaglucosidase Alfa/Miglustat | EXPERIMENTAL | Participants received cipaglucosidase alfa co-administered with miglustat every 2 weeks (Q2W). |
| Alglucosidase Alfa/Placebo | ACTIVE_COMPARATOR | Participants received alglucosidase alfa co-administered with placebo Q2W. |
| Name | Type | Description |
|---|---|---|
| Cipaglucosidase Alfa | BIOLOGICAL | Enzyme Replacement Therapy via intravenous infusion |
| Miglustat | DRUG | Participants received Cipaglucosidase Alfa (ATB200) co-administered with Miglustat(AT2221) |
| Alglucosidase Alfa | BIOLOGICAL | Participants received an IV infusion dose over a 4-hour duration Q2W. |
| Placebo | DRUG | Miglustat matching placebo was administered orally 1 hour prior to alglucosidase alfa infusion Q2W. |
Inclusion Criteria: 1. Male or female subjects (ERT-naïve \[have never received a dose of rhGAA\] or ERT-experienced \[have received rhGAA every 2 weeks for at least 6 months immediately before enrollment, and if ERT dosage has been modified, must have been on the modified dosage for at least 3 mon...