| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01966029 | BMN 110 Phase 3B in Australian Patients | PHASE3 | COMPLETED | 13 | — | — | Jul 1, 2013 | Dec 1, 2016 | Sep 26, 2019 | 5 | Australia |
The analyses of safety will include all patients who receive any study drug. All safety data will be summarized descriptively. All AEs will be coded using the Medical Dictionary for Regulatory Activities (MedDRA). The incidence of AEs will be summarized by System Organ Class (SOC), Preferred Term (PT), relationship to study drug, and severity. Patient listings will be provided for SAEs, deaths, and AEs leading to study drug discontinuation, or study withdrawals. All AE summaries will include only treatment-emergent AEs (TEAEs) reported during the study period (AEs that occur after the first study drug infusion). Infusion-associated AEs will be summarized by SOC, PT and severity. The following safety measures will be summarized descriptively: concomitant medications, clinical laboratory tests, vital signs, ECGs, immunogenicity results, analgesic medication use, results from routine physical examinations (including standard neurologic examinations), and cervical spine imaging.
| Arm | Type | Description |
|---|---|---|
| Treatment | EXPERIMENTAL | All patients receive treatment with BMN 110 |
| Name | Type | Description |
|---|---|---|
| BMN 110 | DRUG | All pateints receive treatment with BMN 110 |
Inclusion Criteria: 1. Diagnosed with MPS IVA as confirmed by a documented GALNS enzymatic test (GALNS activity in affected range, beta-galactosidase and a second lysosomal sulfatase activity within normal range). 2. Age 12 months or older. 3. Willing and able to provide written, signed informed co...