Calendario›BMRN›Vimizim® · MARS

Vimizim® · MARS

A Multicenter, Multinational, Observational Morquio A Registry Study (MARS)

Ensayo de BIOMARIN PHARMACEUTICAL INC en Mucopolysaccharidosis IV Type A · Morquio A Syndrome · MPS IVA (del registro, en inglés).

Más ensayos de: Síndrome de Morquio A.

Fase

no aplica

Estado

Completado

Participantes

418

previstos

Centros

79

Fin del objetivo primario

feb 2024

fecha real

Qué significa cada fase y cada estado.

Estudio observacional. Comenzó en sep 2014.

Qué mide

Safety of Vimizim treated patients as measured by the incidences of Adverse Events, Serious Adverse Events, and immunology tests (del registro, en inglés) (10 años)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe objectives of this program are: to characterize and describe the Mucopolysaccharidosis IV type A (MPS IVA) population as a whole, including the heterogeneity, progression, and natural history of MPS IVA; to evaluate the long-term effectiveness and safety of Vimizim®, including, but not limited to, the occurrence of serious hypersensitivity reactions, anaphylaxis, and changes in antibody status; to help the medical community with the development of recommendations for monitoring MPS IVA patients and reports on patient outcomes to optimize patient care; to collect data on other treatment paradigms, and evaluate the prevalences of their use and their effectiveness; to characterize the effects and safety of Vimizim treatment 5 years from enrollment in the Registry for patients under 5 years of age; to monitor pregnancy exposure, including maternal, neonatal, and infant outcomes; and to monitor patients who have completed the MOR-005 and MOR-007 clinical trials. These patients will be encouraged to enroll in the applicable Registry Substudy and will be monitored using the MOR-005 and MOR-007 assessment schedules, respectively.

Más ensayos de BMRN

Todos los de BMRN, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT02294877), actualizada en mar 2024.