Calendario›RARE›UX003

UX003

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

Ensayo de Ultragenyx Pharmaceutical Inc. en MPS 7 · Sly Syndrome · Mucopolysaccharidosis · MPS VII (del registro, en inglés).

Fase

Fase 3

Estado

Completado

Participantes

12

previstos

Centros

4

Fin del objetivo primario

may 2016

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en dic 2014.

Qué mide

European Union (EU) and Rest of World: Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate (DS) at UX003 Treatment Week 24 (Baseline (defined as the average of all assessments prior to or on the date of cross-over to active treatment with UX003) to 24 weeks of UX003 study drug treatment) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe 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).

Más ensayos de RARE

Todos los de RARE, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT02230566), actualizada en jul 2020.