At a glance
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A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7
In Brief
A Phase 3 clinical trial evaluating UX003 and Placebo for MPS 7 and 3 related conditions. Completed, enrolled 12 participants across 4 sites.
Detailed Summary
The Phase 3 study will use a novel randomized, intra-subject placebo-controlled, single crossover design, referred to as Blind Start, to evaluate the safety and efficacy of UX003. The Blind Start is a novel design whereby participants will be randomized to 1 of 4 groups, each representing a different treatment sequence, and will cross over to UX003 at different pre-defined time points in a blinded manner. All groups will receive a minimum of 24 weeks treatment with 4 mg/kg UX003 every other week (QOW).
Study Details
Timeline
Interventions
UX003 is a sterile concentrate formulation of rhGUS for intravenous infusion
Placebo consisting of the UX003 formulation buffer (without rhGUS)