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MPS 7 clinical trials

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NCT ID: NCT03604835 Recruiting - Clinical trials for Mucopolysaccharidosis VII

Mucopolysaccharidosis VII Disease Monitoring Program

Start date: January 29, 2018
Phase:
Study type: Observational

The objectives of this study are to characterize MPS VII disease presentation and progression and assess long-term effectiveness and safety, including hypersensitivity reactions and immunogenicity of vestronidase alfa.

NCT ID: NCT02230566 Completed - Clinical trials for Mucopolysaccharidosis

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

Start date: December 2014
Phase: Phase 3
Study type: Interventional

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).