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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01275066 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | PHASE3 | COMPLETED | 177 | — | — | Feb 1, 2011 | Aug 1, 2012 | Jul 7, 2014 | 28 | United States, Argentina +15 |
| Arm | Type | Description |
|---|---|---|
| Placebo | PLACEBO_COMPARATOR | - |
| BMN 110 Weekly | EXPERIMENTAL | - |
| BMN 110 Every Other Week | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| BMN 110 Weekly | DRUG | BMN 110 Weekly: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours once a week. |
| Placebo | DRUG | Intravenous infusion of placebo solution at a volume equivalent to that needed for 2.0 mg/kg dose of BMN 110 administered over a period of approximately 4 hours once a week. |
| BMN 110 Every Other Week | DRUG | BMN 110 Every Other Week: Intravenous infusion of BMN 110 at a dose of 2.0 mg/kg administered over a period of approximately 4 hours every other week and infusions of placebo on alternating weeks. |
Inclusion Criteria: * At least 5 years of age. * Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA. * Willing and able to provide written, s...