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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00025896 | Safety and Efficacy of Recombinant Human Acid Alpha-Glucosidase in the Treatment of Classical Infantile Pompe Disease | PHASE2 | COMPLETED | 8 | — | — | May 1, 2001 | Sep 1, 2002 | Nov 13, 2014 | 1 | United States |
| Name | Type | Description |
|---|---|---|
| recombinant human acid alpha-glucosidase (rhGAA) | DRUG | - |
Inclusion Criteria: * Clinical diagnosis of Classical Infantile Pompe Disease * endogenous GAA activity \< 1.0% * cardiomegaly * cardiomyopathy * CRIM (+) * ability to comply with the clinical protocol which will require extensive clinical evaluations Exclusion Criteria: * respiratory insufficien...