Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Iduronate-2-sulfatase enzyme replacement therapy · 1 trial · 1 indication
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...
Iduronate-2-sulfatase enzyme replacement therapy is used for Mucopolysaccharidosis II (MPS II), a rare genetic disorder. It is being developed as a treatment for this condition and is currently in Phase 2 clinical development.
Iduronate-2-sulfatase enzyme replacement therapy is being developed by Takeda Pharmaceutical Company Limited, which is listed on the stock exchange under the ticker TAK.
Iduronate-2-sulfatase enzyme replacement therapy is in Phase 2 clinical development. It is an investigational therapy and has not been approved by regulatory authorities. One Phase 2 trial has been completed.
Iduronate-2-sulfatase enzyme replacement therapy has one completed clinical trial, identified as NCT00069641. This Phase 2 study enrolled 96 male participants aged 5 years and older with Mucopolysaccharidosis II, and was conducted in the United States, Brazil, Germany, and the United Kingdom.
Iduronate-2-sulfatase enzyme replacement therapy is a form of enzyme replacement therapy for Mucopolysaccharidosis II. The specific brand name or alternative name for this therapy is not provided in the available information.