RESUMO
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 .
Assuntos
Miosite Ossificante , Ossificação Heterotópica , Adulto , Humanos , Miosite Ossificante/tratamento farmacológico , Miosite Ossificante/patologia , Tomografia por Emissão de Pósitrons combinada à Tomografia Computadorizada , Ossificação Heterotópica/patologiaAssuntos
Aclimatação , Atletas , Calor Extremo/efeitos adversos , Golpe de Calor/etiologia , Golpe de Calor/prevenção & controle , Condicionamento Físico Humano , Segurança , Feminino , Frequência Cardíaca , Golpe de Calor/fisiopatologia , Humanos , Masculino , Medicina Esportiva , Banho a Vapor , Sudorese , TelemetriaAssuntos
Biodiversidade , Conservação dos Recursos Naturais/legislação & jurisprudência , Política Ambiental/legislação & jurisprudência , Reforma Urbana , Animais , Animais Selvagens/virologia , COVID-19/prevenção & controle , COVID-19/transmissão , China , Cidades/economia , Cidades/estatística & dados numéricos , Abastecimento de Alimentos/legislação & jurisprudência , Inquéritos e Questionários , Reforma Urbana/economia , Zoonoses Virais/epidemiologia , Zoonoses Virais/prevenção & controleRESUMO
An amendment to this paper has been published and can be accessed via a link at the top of the paper.