NCT02230566

A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: Ultragenyx Pharmaceutical Inc · Industry
• Sex: All
• Age: 5 Years – 35 Years
• Healthy volunteers: Not accepted

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).

Conditions

• MPS 7
• Sly Syndrome
• Mucopolysaccharidosis
• MPS VII

Interventions

Type	Name	Description
DRUG	UX003	UX003 is a sterile concentrate formulation of rhGUS for intravenous infusion
OTHER	Placebo	Placebo consisting of the UX003 formulation buffer (without rhGUS)

Timeline

• Start date: 2014-12-01
• Primary completion: 2016-05-01
• Completion: 2016-05-01
• First posted: 2014-09-03
• Last updated: 2020-07-30
• Results posted: 2018-02-19

Locations

4 sites across 1 country: United States