At a glance
ClinicalIndex Comparison Record- ✓Confirmed MPS II diagnosis based on clinical criteria (hepatosplenomegaly, dysostosis multiplex on imaging, valvular heart disease, or obstructive pulmonary disease) and biochemical evidence (iduronate-2-sulfatase deficiency ≤10% of lower limit of normal range; normal activity of another sulfatase)
- ✓Age 5–25 years, male
- ✓Forced vital capacity <80% of predicted at baseline
- ✓Able to perform reproducible pulmonary function testing by spirometry
- ✕Receipt of investigational therapy within 60 days prior to enrollment
- ✕Tracheostomy
- ✕History of bone marrow or cord blood transplant
- ✕Known hypersensitivity to iduronate-2-sulfatase or its components
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II
In Brief
A Phase 3 clinical trial evaluating Iduronate-2-sulfatase enzyme replacement therapy, iduronate-2-sulfatase enzyme replacement therapy, and 1 other intervention for Mucopolysaccharidosis II. Completed, enrolled 96 participants across 9 sites in 4 countries.
Detailed Summary
The purpose of this study is to determine whether the administration of iduronate-2-sulfatase enzyme in a weekly or every other week therapy frequency is safe and efficacious in patients with MPS II.
Study Details
Timeline
Interventions
Patients will receive weekly infusions of idursulfase at a dose of 0.5 mg/kg.
Patients will receive every other week infusions of idursulfase at a dose of 0.5 mg/kg.
Patients will receive weekly infusions of placebo.