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UX003

An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)

Ensayo de Ultragenyx Pharmaceutical Inc. en Mucopolysaccharidosis Type 7 (del registro, en inglés).

Fase

Fase 1/2

Estado

Completado

Participantes

3

previstos

Centros

1

Fin del objetivo primario

jul 2016

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en nov 2013.

Qué mide

Percentage Change From Baseline in Urinary Glycosaminoglycan (uGAG) Dermatan Sulfate (Baseline, Week 14, Week 22, Week 30, Week 38, Week 72, and end of study (up to Week 132)) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroUX003-CL201 is an open-label Phase 1/2 study to assess the safety, efficacy, and dose of UX003 in MPS 7 patients via intravenous (IV) administration every other week (QOW) for 36 weeks with up to an additional 36 weeks from the optional continuation period. Up to 5 participants, who are between 5 and 30 years of age inclusive, will be enrolled and treated with UX003. The initial 12-week treatment period will be followed by a 24-week forced dose titration period to assess the optimal dose. Participants who complete both the initial treatment and forced dose titration periods will continue treatment in a 36- week continuation period.

Más ensayos de RARE

Todos los de RARE, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT01856218), actualizada en dic 2018.