{"id":1097,"date":"2022-09-13T19:56:58","date_gmt":"2022-09-13T19:56:58","guid":{"rendered":"https:\/\/vimizimhcp-dev-001.azurewebsites.net\/en-us\/vimizim\/?page_id=1097"},"modified":"2023-05-03T11:45:18","modified_gmt":"2023-05-03T11:45:18","slug":"study-design","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/study-design\/","title":{"rendered":"Study Design"},"content":{"rendered":"<div id=\"acf-block-643e71965ffe6\" class=\"page-header\">         \n            \n        <div class=\"header-image\" style=\"background-image: url(https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/09\/Derric.png?v=0.46)\"><\/div>\n     \n    <div class=\"header-content\">\n                    <h1>VIMIZIM<sup>\u00ae<\/sup> (elosulfase alfa) efficacy was evaluated in a pivotal phase 3 trial and a long-term extension study<sup>1,2<\/sup><\/h1>\n            <\/div>\n<\/div>\n\n<div id=\"acf-block-643e7196604a3\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-643e719660692\" class=\"block-wysiwyg\">\n            <h2 class=\"h3\">Design of the pivotal MOR-004 study and the long-term MOR-005 extension study<sup>1,2<\/sup><\/h2>\n    <\/div>\n\n<figure id=\"acf-block-643e7196606be\">\n    <div class=\"image image-rounded image-align-left\">\n                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/09\/study-design-1900x1085.png\" alt=\"MOR-004 study and the long-term MOR-005 extension study\" \/>            <\/div>\n    <\/figure>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-643e7196606c9\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-643e719660707\" class=\"block-wysiwyg\">\n            <h2>MOR-005 data analyses<\/h2>\n<ul>\n<li>In addition to the intent-to-treat (ITT) population (N=173), analyses were also performed for an MPP population (n=124)<sup>2<\/sup><\/li>\n<li>The MPP population excluded patients who had orthopedic surgery and\/or missed \u226520% of their scheduled VIMIZIM\u00ae (elosulfase alfa) infusions<sup>2<\/sup><\/li>\n<li>In a post hoc analysis, treatment groups were compared with matched historical controls from the MorCAP natural history study<sup>2<\/sup>\n<ul>\n<li>The ITT population was compared to MorCAP patients who met the phase 3 trial inclusion criteria<\/li>\n<li>The MPP population was compared to a matched MorCAP group that met the phase 3 trial inclusion criteria and also excluded patients who underwent orthopedic surgery within 3 months prior to baseline or during the subsequent 2 years of follow-up<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-643e719660732\" class=\"block boxed-content top-border boxed-content-white\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\n<div id=\"acf-block-643e719660767\" class=\"image-text-block\">\n        <div class=\"content-block\">\n                                                    <p><a class=\"button button-text button-arrow\" href=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/vimizim-safety-and-tolerability\/\" target=\"_self\">Review the safety information for VIMIZIM<\/a><\/p>\n                        <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-643e71966079c\" class=\"block references\">\n    <div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t    \t\t\t    <h4>References:\n<\/h4>\n\t\t\t\t\t\t                <ol>\n                                                                                                                        <li><span>Hendriksz CJ, Burton B, Fleming TR, <em>et al.<\/em> Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. <em>J Inherit Metab Dis<\/em>. 2014;37(6):979-990.\n<\/span><\/li>\n                                                                                                                                                <li><span>Hendriksz CJ, Parini R, AlSayed MD,<em> et al<\/em>. Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome. <em>Mol Genet Metab<\/em>. 2016;119(1-2):131-143.\n<\/span><\/li>\n                                                            <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-1097","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>VIMIZIM\u00ae (elosulfase alfa) | Study Design<\/title>\n<meta name=\"description\" content=\"Examine the study design of the pivotal Phase 3 trial and long-term extension study of VIMIZIM\u00ae (elosulfase alfa) in patients with Moquino A. 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