RESUMEN
Fibrodysplasia ossificans progressiva (FOP) is a rare disease characterized by heterotopic ossification (HO) in connective tissues and painful flare-ups. In the phase 2 LUMINA-1 trial, adult patients with FOP were randomized to garetosmab, an activin A-blocking antibody (n = 20) or placebo (n = 24) in period 1 (28 weeks), followed by an open-label period 2 (28 weeks; n = 43). The primary end points were safety and for period 1, the activity and size of HO lesions. All patients experienced at least one treatment-emergent adverse event during period 1, notably epistaxis, madarosis and skin abscesses. Five deaths (5 of 44; 11.4%) occurred in the open-label period and, while considered unlikely to be related, causality cannot be ruled out. The primary efficacy end point in period 1 (total lesion activity by PET-CT) was not met (P = 0.0741). As the development of new HO lesions was suppressed in period 1, the primary efficacy end point in period 2 was prospectively changed to the number of new HO lesions versus period 1. No placebo patients crossing over to garetosmab developed new HO lesions (0% in period 2 versus 40.9% in period 1; P = 0.0027). Further investigation of garetosmab in FOP is ongoing. ClinicalTrials.gov identifier NCT03188666 .
Asunto(s)
Miositis Osificante , Osificación Heterotópica , Adulto , Humanos , Miositis Osificante/tratamiento farmacológico , Miositis Osificante/patología , Tomografía Computarizada por Tomografía de Emisión de Positrones , Osificación Heterotópica/patologíaAsunto(s)
Aclimatación , Atletas , Calor Extremo/efectos adversos , Golpe de Calor/etiología , Golpe de Calor/prevención & control , Acondicionamiento Físico Humano , Seguridad , Femenino , Frecuencia Cardíaca , Golpe de Calor/fisiopatología , Humanos , Masculino , Medicina Deportiva , Baño de Vapor , Sudoración , TelemetríaAsunto(s)
Biodiversidad , Conservación de los Recursos Naturales/legislación & jurisprudencia , Política Ambiental/legislación & jurisprudencia , Remodelación Urbana , Animales , Animales Salvajes/virología , COVID-19/prevención & control , COVID-19/transmisión , China , Ciudades/economía , Ciudades/estadística & datos numéricos , Abastecimiento de Alimentos/legislación & jurisprudencia , Encuestas y Cuestionarios , Remodelación Urbana/economía , Zoonosis Virales/epidemiología , Zoonosis Virales/prevención & controlRESUMEN
An amendment to this paper has been published and can be accessed via a link at the top of the paper.