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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00104234 | Study of rhASB in Patients With Mucopolysaccharidosis VI | PHASE3 | COMPLETED | 39 | — | — | Feb 1, 2004 | Oct 1, 2006 | Feb 2, 2010 | 1 | United States |
| NCT00067470 | Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI | PHASE3 | COMPLETED | 39 | — | — | Sep 1, 2003 | Mar 1, 2004 | Dec 24, 2009 | 1 | United States |
| NCT00048711 | Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI | PHASE2 | COMPLETED | - | — | — | Mar 1, 2002 | Jun 1, 2006 | Aug 25, 2009 | 1 | United States |
| NCT00048620 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | PHASE1 | COMPLETED | - | — | — | Sep 1, 2000 | Nov 1, 2005 | Nov 3, 2006 | 1 | United States |
Mean change in meters walked in 12 minutes. Mean change is the mean difference between the 12-Minute Walk Test at 96 weeks and that measured before first ever treatment with rhASB. For the rhASB/rhASB group, mean change is calculated for Week 96 - Baseline. For the placebo/rhASB group, mean change is calculated for Week 96 - Week 24.
Number of meters walked in 12 minutes at week 24 minus number of meters walked in 12 minutes at baseline
| Arm | Type | Description |
|---|---|---|
| rhASB/rhASB | OTHER | N-acetylgalactosamine 4-sulfatase |
| Placebo/rhASB | OTHER | - |
| Placebo | PLACEBO_COMPARATOR | - |
| rhASB | ACTIVE_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| N-acetylgalactosamine 4-sulfatase | DRUG | - |
| Placebo/rhASB | DRUG | - |
| Placebo | DRUG | - |
Inclusion Criteria: * Patient consent * Patient must have successfully completed study ASB-03-05, including having received at least 20 of 24 scheduled weekly infusions and having missed no more then two consecutive infusions * If female of childbearing potential, patient must have a negative pregn...