{"id":1060,"date":"2013-09-10T16:38:39","date_gmt":"2013-09-10T14:38:39","guid":{"rendered":"https:\/\/www.aica3.org\/cms\/?p=1060"},"modified":"2022-01-06T10:08:35","modified_gmt":"2022-01-06T09:08:35","slug":"uno-studio-di-fase-i-ii-di-myo-029-in-soggetti-adulti-con-distrofia-muscolare","status":"publish","type":"post","link":"https:\/\/www.aica3.org\/cms\/en\/uno-studio-di-fase-i-ii-di-myo-029-in-soggetti-adulti-con-distrofia-muscolare\/","title":{"rendered":"A Phase I\/II trial of MYO-029 in Adult Subjects with Muscular Dystrophy"},"content":{"rendered":"<div title=\"Pagina 1\">\n<div>\n<div>\n<p>Objective: Myostatin is an endogenous negative regulator of muscle growth and a novel target for muscle diseases. We con- ducted a safety trial of a neutralizing antibody to myostatin, MYO-029, in adult muscular dystrophies (Becker muscular dys- trophy, facioscapulohumeral dystrophy, and limb-girdle muscular dystrophy).\nMethods: This double-blind, placebo-controlled, multinational, randomized study included 116 subjects divided into sequential dose-escalation cohorts, each receiving MYO-029 or placebo (Cohort 1 at 1mg\/kg; Cohort 2 at 3mg\/kg; Cohort 3 at 10mg\/kg; Cohort 4 at 30mg\/kg). Safety and adverse events were assessed by reported signs and symptoms, as well as by physical exam- inations, laboratory results, echocardiograms, electrocardiograms, and in subjects with facioscapulohumeral dystrophy, fundus- copic and audiometry examinations. Biological activity of MYO-029 was assessed through manual muscle testing, quantitative muscle testing, timed function tests, subject-reported outcomes, magnetic resonance imaging studies, dual-energy radiographic absorptiometry studies, and muscle biopsy.<\/p>\n<p>Results: MYO-029 had good safety and tolerability with the exception of cutaneous hypersensitivity at the 10 and 30mg\/kg doses. There were no improvements noted in exploratory end points of muscle strength or function, but the study was not powered to look for efficacy. Importantly, bioactivity of MYO-029 was supported by a trend in a limited number of subjects toward increased muscle size using dual-energy radiographic absorptiometry and muscle histology.<\/p>\n<p>Interpretation: This trial supports the hypothesis that systemic administration of myostatin inhibitors provides an adequate safety margin for clinical studies. Further evaluation of more potent myostatin inhibitors for stimulating muscle growth in muscular dystrophy should be considered.<\/p>\n<p><a href=\"https:\/\/www.aica3.org\/cms\/wp-content\/uploads\/2013\/09\/A-Phase-III-trial-of-MYO-029-in-Adult-Subjects-with-Muscular-Dystrophy.pdf\">A Phase I\/II trial of MYO-029 in Adult Subjects with Muscular Dystrophy<\/a><\/p>\n<\/div><\/div>\n<\/p><\/div>\n<\/p><\/div>","protected":false},"excerpt":{"rendered":"<p>Obiettivo: La miostatina \u00e8 un regolatore negativo endogeno della crescita muscolare e un nuovo bersaglio per le malattie muscolari. Abbiamo condotto uno studio sulla sicurezza di un anticorpo neutralizzante della miostatina, MYO-029, nelle distrofie muscolari adulte (distrofia muscolare Becker, distrofia facioscapolo-omerale e distrofia muscolare del cingolo degli arti): Questo studio in doppio cieco, controllato con [&hellip;]<\/p>\n","protected":false},"author":4,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":""},"categories":[34,33],"tags":[],"class_list":["post-1060","post","type-post","status-publish","format-standard","hentry","category-articoli-scientifici","category-ricerca-e-pubblicazioni"],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Uno studio di fase I\/II di MYO-029 in soggetti adulti con distrofia muscolare - AICa3 - ETS - Associazione Italiana Calpaina 3<\/title>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/www.aica3.org\/cms\/en\/uno-studio-di-fase-i-ii-di-myo-029-in-soggetti-adulti-con-distrofia-muscolare\/\" \/>\n<meta property=\"og:locale\" content=\"en_GB\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Uno studio di fase I\/II di MYO-029 in soggetti adulti con distrofia muscolare - AICa3 - ETS - Associazione Italiana Calpaina 3\" \/>\n<meta property=\"og:description\" content=\"Obiettivo: La miostatina \u00e8 un regolatore negativo endogeno della crescita muscolare e un nuovo bersaglio per le malattie muscolari. 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