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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01230801 | Safety/Tolerability/Pharmacokinetic (PK)/Pharmacodynamics (PD) Study of BMN701 in Patients With Late-Onset Pompe Disease | PHASE1 | COMPLETED | 22 | — | — | Jan 17, 2011 | Mar 6, 2013 | Jun 11, 2018 | 10 | United States, Australia +3 |
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
| Arm | Type | Description |
|---|---|---|
| BMN 701 | EXPERIMENTAL | IV infusion |
| Name | Type | Description |
|---|---|---|
| BMN 701 | BIOLOGICAL | GILT-tagged recombinant human GAA |
Inclusion criteria: * Patient has been diagnosed with Pompe Disease prior to or during the screening period based on 2 GAA gene mutations and either: endogenous GAA activity \<75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed in cultured skin fibroblas...