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VISTA

VIrtual STudy in Achondroplasia for the US (VISTA)

Ensayo de BIOMARIN PHARMACEUTICAL INC en Acondroplasia.

Más ensayos de: Acondroplasia.

Fase

no aplica

Estado

Reclutando pacientes

Participantes

170

previstos

Centros

8

Fin del objetivo primario

feb 2028

estimado por el promotor

Qué significa cada fase y cada estado.

Estudio observacional. Comenzó en feb 2023.

Qué mide

Height (Retrospective data and prospective data for up to 5 years starting from time of study enrolment) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis is an observational study of individuals with achondroplasia in the United States. The primary study population consists of pediatric individuals treated and untreated with VOXZOGO™. Study enrollment started in February 2023. The projected total duration of the study is approximately 5 years at minimum from start of study recruitment in February 2023, with the duration of individual prospective follow-up differing depending on the time of enrollment. The study duration may be extended based on decisions by the study sponsor. Data will be collected in two formats for the primary pediatric study population: 1. Participant-mediated access to electronic health records(including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. . 2. Primary data collection of Clinical Outcome Assessments (COAs) and questionnaire data. Data will be collected in the following format for the adult cohort: \- Participant-mediated access to electronic health records (including medical imaging, when available) which will enable retrospective and prospective collection of secondary data reflecting real-life treatment use and clinical care. The primary study population will include approximately 150 pediatric individuals with achondroplasia regardless of their treatment status with VOXZOGO™. Individuals may change status from untreated to treated during the prospective period of the study (or vice versa) however they will only be counted once, based on their treated status at the point of enrollment. The secondary study population will include 20 adolescent and adult participants.

Más ensayos de BMRN

Todos los de BMRN, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT06168201), actualizada en jun 2026.