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Cardiac Assistive Device

Natural History of Danon Disease

Ensayo de ROCKET PHARMACEUTICALS, INC. en Danon Disease (del registro, en inglés).

Fase

no aplica

Estado

Completado

Participantes

59

previstos

Centros

15

Fin del objetivo primario

oct 2023

fecha real

Qué significa cada fase y cada estado.

Estudio observacional. Comenzó en sep 2022.

Qué mide

Cardiac Manifestations- LVPWd (Retrospective minimum of 6 months) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThis is a multicenter, international, non-interventional, natural history study designed to collect longitudinal retrospective clinical information on patients with Danon disease (DD). This study is composed of 2 parts: * Feasibility study: to identify participating sites, assess site and team capabilities, confirm the site and investigator qualification for taking part in the study, * Retrospective chart review: Data will be collected retrospectively by means of a chart review of living and/or deceased DD patients with a confirmed lysosome associated membrane protein-2 gene (LAMP2) mutation, and, * For living patients (who have not undergone heart transplantation or placement of a cardiac assist device), availability of at least 6-month cardiology follow-up data, * For living patients who underwent heart transplantation or placement of a cardiac assist device, and for deceased patients, at least 1 MRI or echo assessment prior to heart transplantation/cardiac assist device placement or death.

Más ensayos de RCKT

Todos los de RCKT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT05548855), actualizada en nov 2023.