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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT00069641 | Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) | PHASE2 | COMPLETED | 96 | — | — | Sep 18, 2003 | Mar 16, 2005 | Jun 10, 2021 | 9 | United States, Brazil +2 |
The 2-component composite variable consists of the sum of the ranked changes from baseline to Week 53 for percent predicted Forced Vital Capacity (FVC) and 6-Minute Walking Test (6MWT) total distance walked. For the 2 treatment groups being compared, ranking occurred within the comparison treatment groups combined (idursulfase weekly and placebo treatment groups). These comparison groups were pooled and ranked for each component separately. Within each component (% predicted FVC, 6MWT), the change from baseline was then ranked. The lowest change value was assigned a rank of 1, the next lowest a rank of 2, etc. The composite score for each participant was the sum of the 2 ranked scores corresponding to the 2 individual components (% predicted FVC and 6MWT) for each participant. Thus, the greater the composite score (greater the sum of the ranks of the changes from baseline, where the lowest change was ranked as 1), the greater the improvement.
| Arm | Type | Description |
|---|---|---|
| Idursulfase weekly (0.5 mg/kg) | EXPERIMENTAL | - |
| Idursulfase every other week (0.5 mg/kg) | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | - |
| Name | Type | Description |
|---|---|---|
| Iduronate-2-sulfatase enzyme replacement therapy | BIOLOGICAL | Patients will receive weekly infusions of idursulfase at a dose of 0.5 mg/kg. |
| Placebo | BIOLOGICAL | Patients will receive weekly infusions of placebo. |
Inclusion Criteria: To be eligible to participate in this study, patients must meet the following inclusion criteria prior to enrollment: 1. The diagnosis of MPS II will be determined by the investigator based upon both clinical and biochemical criteria. 2. All patients must have at least one of t...