Conditions: MPS 7 Sly Syndrome Mucopolysaccharidosis MPS VII
Keywords: MPS 7 Sly Syndrome Mucopolysaccharidosis MPS VII Enzyme Replacement Therapy Mucopolysaccharidosis type 7 rare disease lysosomal storage disease metabolic disorder
Allocation: RANDOMIZED | Intervention model: CROSSOVER
Enrollment: 12
Healthy volunteers: no

Brief 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).

Detailed description:

Arms:
arm 1: 4 mg/kg UX003 QOW through Week 46 arm 2: Placebo QOW for the first 8 weeks followed by 4 mg/kg UX003 QOW through Week 46 arm 3: Placebo QOW for the first 16 weeks followed by 4 mg/kg UX003 QOW through Week 46 arm 4: Placebo QOW for the first 24 weeks followed by 4 mg/kg UX003 QOW through Week 46

Interventions: intervention 1: UX003 intervention 2: Placebo
Intervention descriptions: intervention 1: UX003 is a sterile concentrate formulation of rhGUS for intravenous infusion intervention 2: Placebo consisting of the UX003 formulation buffer (without rhGUS)

Protocol document: