{"id":440,"date":"2022-08-10T14:19:22","date_gmt":"2022-08-10T18:19:22","guid":{"rendered":"https:\/\/campsiitestudy.com\/para-profesionales-sanitarios\/"},"modified":"2023-06-06T16:35:33","modified_gmt":"2023-06-06T20:35:33","slug":"para-profesionales-sanitarios","status":"publish","type":"page","link":"https:\/\/campsiitestudy.com\/es\/para-profesionales-sanitarios\/","title":{"rendered":"Para profesionales sanitarios"},"content":{"rendered":"\t\t<div data-elementor-type=\"wp-page\" data-elementor-id=\"440\" class=\"elementor elementor-440 elementor-11\" data-elementor-post-type=\"page\">\n\t\t\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-ad3745b elementor-section-stretched subpage-hero elementor-section-full_width elementor-section-height-default elementor-section-height-default\" data-id=\"ad3745b\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;stretch_section&quot;:&quot;section-stretched&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-no\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-74af6b8\" data-id=\"74af6b8\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-f89cf04 elementor-widget__width-auto elementor-absolute header-illustration cloud-1 elementor-widget elementor-widget-image\" data-id=\"f89cf04\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img decoding=\"async\" width=\"245\" height=\"94\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/cloud-1-1.svg\" class=\"attachment-full size-full wp-image-320\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-7581d9f elementor-widget__width-auto elementor-absolute header-illustration cloud-2 elementor-widget elementor-widget-image\" data-id=\"7581d9f\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img decoding=\"async\" width=\"212\" height=\"89\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/cloud-2.svg\" class=\"attachment-full size-full wp-image-293\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-8ecfaa0 elementor-widget__width-auto elementor-absolute header-illustration sun elementor-widget elementor-widget-image\" data-id=\"8ecfaa0\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;,&quot;motion_fx_motion_fx_scrolling&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_effect&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_direction&quot;:&quot;negative&quot;,&quot;motion_fx_translateY_speed&quot;:{&quot;unit&quot;:&quot;px&quot;,&quot;size&quot;:1,&quot;sizes&quot;:[]},&quot;motion_fx_translateY_affectedRange&quot;:{&quot;unit&quot;:&quot;%&quot;,&quot;size&quot;:&quot;&quot;,&quot;sizes&quot;:{&quot;start&quot;:0,&quot;end&quot;:100}},&quot;motion_fx_devices&quot;:[&quot;desktop&quot;,&quot;tablet&quot;,&quot;mobile&quot;]}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img fetchpriority=\"high\" decoding=\"async\" width=\"224\" height=\"224\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/sun.svg\" class=\"attachment-full size-full wp-image-302\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-f72ee61 elementor-widget__width-auto elementor-absolute header-illustration cloud-3 elementor-widget elementor-widget-image\" data-id=\"f72ee61\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"245\" height=\"94\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/cloud-3.svg\" class=\"attachment-full size-full wp-image-296\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-954235d elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"954235d\" data-element_type=\"section\" data-e-type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-66 elementor-inner-column elementor-element elementor-element-b38efda\" data-id=\"b38efda\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-5b3e9e0 elementor-widget elementor-widget-shortcode\" data-id=\"5b3e9e0\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"shortcode.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<div class=\"breadcrumb\" role=\"navigation\" aria-label=\"breadcrumbs\"><div class=\"wrap\"><span class=\"breadcrumb-link-wrap\"><a class=\"breadcrumb-link\" href=\"https:\/\/campsiitestudy.com\/es\/\"><span class=\"breadcrumb-link-text-wrap\"><i class=\"fa-solid fa-tent\"><\/i><span class=\"screen-reader-text\">Home<\/span><\/span><\/a><meta ><\/span><\/div><\/div>\t\t<div class=\"elementor-shortcode\"><\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-05127fe elementor-widget elementor-widget-text-editor\" data-id=\"05127fe\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>CAMPSIITE\u2122 es un estudio multic\u00e9ntrico, abierto, de un solo grupo, de fase I\/II\/III en el que se inscribi\u00f3 a ni\u00f1os varones con MPS II neuronop\u00e1tica, de 4 meses de edad hasta los 5 a\u00f1os de edad, para evaluar la eficacia, seguridad y farmacodin\u00e1mica (FD) de la terapia g\u00e9nica RGX-121.<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-33 elementor-inner-column elementor-element elementor-element-cf34daf\" data-id=\"cf34daf\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap\">\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<div class=\"elementor-element elementor-element-bcb3433 elementor-widget__width-auto elementor-absolute header-illustration tree-2 elementor-widget elementor-widget-image\" data-id=\"bcb3433\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;,&quot;motion_fx_motion_fx_scrolling&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_effect&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_speed&quot;:{&quot;unit&quot;:&quot;px&quot;,&quot;size&quot;:1,&quot;sizes&quot;:[]},&quot;motion_fx_translateY_affectedRange&quot;:{&quot;unit&quot;:&quot;%&quot;,&quot;size&quot;:&quot;&quot;,&quot;sizes&quot;:{&quot;start&quot;:0,&quot;end&quot;:100}},&quot;motion_fx_devices&quot;:[&quot;desktop&quot;,&quot;tablet&quot;,&quot;mobile&quot;]}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"300\" height=\"392\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/tree-2.svg\" class=\"attachment-full size-full wp-image-305\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-cdc41b7 elementor-widget__width-auto elementor-absolute header-illustration tree-1 elementor-widget elementor-widget-image\" data-id=\"cdc41b7\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;,&quot;motion_fx_motion_fx_scrolling&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_effect&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_speed&quot;:{&quot;unit&quot;:&quot;px&quot;,&quot;size&quot;:1,&quot;sizes&quot;:[]},&quot;motion_fx_translateY_affectedRange&quot;:{&quot;unit&quot;:&quot;%&quot;,&quot;size&quot;:&quot;&quot;,&quot;sizes&quot;:{&quot;start&quot;:0,&quot;end&quot;:100}},&quot;motion_fx_devices&quot;:[&quot;desktop&quot;,&quot;tablet&quot;,&quot;mobile&quot;]}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"200\" height=\"261\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/tree-1.svg\" class=\"attachment-full size-full wp-image-299\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-0f0b652 elementor-widget__width-auto elementor-absolute header-illustration tree-4 elementor-widget elementor-widget-image\" data-id=\"0f0b652\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_position&quot;:&quot;absolute&quot;,&quot;motion_fx_motion_fx_scrolling&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_effect&quot;:&quot;yes&quot;,&quot;motion_fx_translateY_speed&quot;:{&quot;unit&quot;:&quot;px&quot;,&quot;size&quot;:1,&quot;sizes&quot;:[]},&quot;motion_fx_translateY_affectedRange&quot;:{&quot;unit&quot;:&quot;%&quot;,&quot;size&quot;:&quot;&quot;,&quot;sizes&quot;:{&quot;start&quot;:0,&quot;end&quot;:100}},&quot;motion_fx_devices&quot;:[&quot;desktop&quot;,&quot;tablet&quot;,&quot;mobile&quot;]}\" data-widget_type=\"image.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<img loading=\"lazy\" decoding=\"async\" width=\"400\" height=\"523\" src=\"https:\/\/campsiitestudy.com\/wp-content\/uploads\/2022\/08\/tree-4.svg\" class=\"attachment-full size-full wp-image-311\" alt=\"\" \/>\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-b9cf9ea summary-key-points elementor-section-stretched elementor-section-boxed elementor-section-height-default elementor-section-height-default elementor-invisible\" data-id=\"b9cf9ea\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;,&quot;stretch_section&quot;:&quot;section-stretched&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-648d85e card\" data-id=\"648d85e\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-ed27cc6 elementor-widget elementor-widget-text-editor\" data-id=\"ed27cc6\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Resumen de los puntos clave<\/h3>\n<ul>\n<li>Los datos emergentes de la evoluci\u00f3n natural destacan la urgencia de tratar pronto la MPS II neuronop\u00e1tica.<\/li>\n<li>Los datos provisionales de la parte en fase I\/II de CAMPSIITE demuestran que RGX-121 se ha tolerado bien a la fecha del 1 de agosto de 2022. Los datos provisionales del nivel de dosis seleccionado para la expansi\u00f3n fundamental (cohorte 3) mostraron biomarcadores que se acercaban a los niveles normales de GAG.<sup>1<\/sup> <\/li>\n<li>Los datos disponibles de las cohortes 1 y 2 mostraron actividad del SNC hasta 2 a\u00f1os despu\u00e9s de la administraci\u00f3n de RGX-121, como lo demuestran las mejoras en la funci\u00f3n del neurodesarrollo y los resultados notificados por el cuidador.<sup>2<\/sup><\/li>\n<li>CAMPSIITE est\u00e1 actualmente inscribiendo a pacientes pedi\u00e1tricos con MPS II neuronop\u00e1tica de 4 meses a 5 a\u00f1os de edad al nivel de dosis fundamental para evaluar los niveles de GAG en el l\u00edquido cefalorraqu\u00eddeo (LCR) y evaluar el efecto de RGX-121 sobre la funci\u00f3n del neurodesarrollo.<\/li>\n<li>Si tiene un paciente menor de 5 a\u00f1os que tiene o puede tener MPS II neuronop\u00e1tica, p\u00f3ngase en contacto con un centro del estudio o p\u00f3ngase en contacto con <a href=\"mailto:medinfo@regenxbio.com\">medinfo@regenxbio.com<\/a> para obtener m\u00e1s informaci\u00f3n.<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-6dab7ea elementor-section-stretched light-green-row natural-data-row elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"6dab7ea\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;stretch_section&quot;:&quot;section-stretched&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-1855383\" data-id=\"1855383\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-cfa7936 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"cfa7936\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h2>Datos de la historia natural de la MPS II<\/h2>\n<p>Un an\u00e1lisis de los datos de la historia natural de 32 pacientes (intervalo de edad en la primera evaluaci\u00f3n: de 1 a 117 meses) proporciona una imagen completa y multidominio del desarrollo de la MPS II neuronop\u00e1tica.<\/p>\n<p>Los datos demuestran que:<\/p>\n<ul>\n<li>La adquisici\u00f3n de habilidades de desarrollo se ralentiza en todas las Escalas Mullen de Aprendizaje Temprano (Mullen Scales of Early Learning &#8211; MSEL) cuando se compara con el desarrollo entre compa\u00f1eros (fase de retraso) y que la escala del lenguaje expresivo proporciona la indicaci\u00f3n m\u00e1s temprana de retraso.<sup>3<\/sup><\/li>\n<li>Las trayectorias medias de la puntuaci\u00f3n equivalente a la edad (AEq) del desarrollo comienzan a desviarse ya a los 12.8 meses.<sup>3<\/sup><\/li>\n<li>La tasa de adquisici\u00f3n de habilidades se ralentiza al principio del desarrollo y el retraso se vuelve m\u00e1s sustancial con el tiempo.<sup>3<\/sup><\/li>\n<\/ul>\n<p>Los datos del an\u00e1lisis de la historia natural se presentaron como p\u00f3ster en la reuni\u00f3n anual de 2022 de la Society for the Study of Inborn Errors of Metabolism &#8211; SSIEM (Sociedad para el Estudio de los Errores Innatos del Metabolismo)<sup>3<\/sup>. <a href=\"\/wp-content\/uploads\/2022\/12\/Escolar-MPS-II-Natural-History-n32-SSIEM22-POSTER-vF.pdf\">Haga clic aqu\u00ed para ver el p\u00f3ster<\/a>.<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<section class=\"elementor-section elementor-top-section elementor-element elementor-element-3ce4066 elementor-section-stretched elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"3ce4066\" data-element_type=\"section\" data-e-type=\"section\" data-settings=\"{&quot;stretch_section&quot;:&quot;section-stretched&quot;}\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-100 elementor-top-column elementor-element elementor-element-35384f8\" data-id=\"35384f8\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-993e849 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"993e849\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h2>Oportunidades de inscripci\u00f3n actuales<\/h2>\n<p>REGENXBIO est\u00e1 inscribiendo a ni\u00f1os varones con MPS II neuronop\u00e1tica, de 4 meses hasta 5 a\u00f1os de edad, en el estudio CAMPSIITE. Los objetivos principales de este ensayo fundamental son evaluar los GAG del l\u00edquido cefalorraqu\u00eddeo (LCR) y el efecto de RGX-121 en la funci\u00f3n del neurodesarrollo medida por las escalas Bayley de desarrollo infantil y ni\u00f1os peque\u00f1os, 3.a edici\u00f3n (BSID-III) o las Escalas Mullen de Aprendizaje Temprano (MSEL) en la MPS II neuronop\u00e1tica pedi\u00e1trica. Tambi\u00e9n se evaluar\u00e1n la seguridad, los resultados notificados por el cuidador y los par\u00e1metros de la resonancia magn\u00e9tica.  <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-aad6934 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"aad6934\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Elegibilidad<\/h3>\n<p>A continuaci\u00f3n se muestran los criterios de elegibilidad para el ensayo CAMPSIITE. Para obtener una lista m\u00e1s completa de los criterios de elegibilidad, una lista de las ubicaciones de los centros del estudio CAMPSIITE y m\u00e1s informaci\u00f3n, visite <a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\" target=\"_blank\" rel=\"noopener\" data-wplink-edit=\"true\">ClinicalTrials.gov<\/a> (identificador n.\u00ba NCT03566043). <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-6d4565f enrollment-chart elementor-invisible elementor-widget elementor-widget-html\" data-id=\"6d4565f\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"html.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t<table>\n    <tr>\n        <th>\n Claves de los criterios de elegibilidad\n        <\/th>\n        <td>\n            <p>Los participantes elegibles deben ser varones de entre 4 meses y 5 a\u00f1os de edad el d\u00eda 1 del ensayo y tener un diagn\u00f3stico documentado de MPS II neuronop\u00e1tica. Para obtener una lista completa de los criterios de elegibilidad e informaci\u00f3n adicional, visite <a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\" target=\"_blank\">clinicaltrials.gov<\/a>. <\/p>\n        <\/td>\n    <\/tr>\n<\/table>\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-ab81566 elementor-widget elementor-widget-text-editor\" data-id=\"ab81566\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p><i>Si se desconoce el fenotipo de MPS II de su paciente, puede derivar a su paciente a un centro del estudio para su evaluaci\u00f3n.<\/i><\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-5111602 ct-callout elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"5111602\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<p>Si tiene alg\u00fan paciente en su consulta que pueda ser elegible para participar en el estudio CAMPSIITE, le animamos a que se ponga en contacto con un centro del estudio.&nbsp;<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-c61a55a elementor-widget-divider--view-line elementor-invisible elementor-widget elementor-widget-divider\" data-id=\"c61a55a\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeIn&quot;,&quot;_animation_delay&quot;:150}\" data-widget_type=\"divider.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t<div class=\"elementor-divider\">\n\t\t\t<span class=\"elementor-divider-separator\">\n\t\t\t\t\t\t<\/span>\n\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<div class=\"elementor-element elementor-element-6670b26 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"6670b26\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Criterios de valoraci\u00f3n principales del estudio<\/h3>\n<p>Se evaluar\u00e1 a los participantes (seguridad y eficacia) durante dos a\u00f1os despu\u00e9s del tratamiento con RGX-121, luego se les animar\u00e1 a participar en un estudio de seguimiento a largo plazo. Los criterios de valoraci\u00f3n principales de la expansi\u00f3n fundamental son: <\/p>\n<ul>\n<li>Niveles de GAG en el LCR<\/li>\n<li>Funci\u00f3n del neurodesarrollo medida por las escalas Bayley de desarrollo infantil y ni\u00f1os peque\u00f1os, 3.a edici\u00f3n (BSID-III) o las Escalas Mullen de Aprendizaje Temprano (MSEL)<\/li>\n<\/ul>\n<p>Para obtener m\u00e1s informaci\u00f3n sobre el ensayo, p\u00f3ngase en contacto con <a href=\"mailto:medinfo@regenxbio.com\">medinfo@regenxbio.com<\/a> o visite <a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\" data-wplink-edit=\"true\">ClinicalTrials.gov<\/a> (identificador n.\u00ba NCT03566043).<\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t<section class=\"elementor-section elementor-inner-section elementor-element elementor-element-08202da callout-row elementor-section-boxed elementor-section-height-default elementor-section-height-default\" data-id=\"08202da\" data-element_type=\"section\" data-e-type=\"section\">\n\t\t\t\t\t\t<div class=\"elementor-container elementor-column-gap-default\">\n\t\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-c0b1bb9 card elementor-invisible\" data-id=\"c0b1bb9\" data-element_type=\"column\" data-e-type=\"column\" data-settings=\"{&quot;animation&quot;:&quot;fadeInUp&quot;}\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-8f11628 elementor-widget elementor-widget-text-editor\" data-id=\"8f11628\" data-element_type=\"widget\" data-e-type=\"widget\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Inscripci\u00f3n actual<\/h3><p>Remita a su paciente a un centro del estudio para su evaluaci\u00f3n<\/p><p><a class=\"button\" href=\"https:\/\/campsiitestudy.com\/es\/#campsiite-locations\">Ubicaciones de centros de CAMPSIITE\u2122<\/a><\/p>\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t<div class=\"elementor-column elementor-col-50 elementor-inner-column elementor-element elementor-element-6d03f64 card\" data-id=\"6d03f64\" data-element_type=\"column\" data-e-type=\"column\">\n\t\t\t<div class=\"elementor-widget-wrap elementor-element-populated\">\n\t\t\t\t\t\t<div class=\"elementor-element elementor-element-546eb56 elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"546eb56\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;,&quot;_animation_delay&quot;:150}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>P\u00f3ngase en contacto con nosotros<\/h3>\n<p>Para obtener m\u00e1s informaci\u00f3n sobre nuestros estudios para pacientes con MPS II, p\u00f3ngase en contacto con nosotros en <a href=\"mailto:medinfo@regenxbio.com\">medinfo@regenxbio.com<\/a>. <\/p>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<div class=\"elementor-element elementor-element-96dda1d references elementor-invisible elementor-widget elementor-widget-text-editor\" data-id=\"96dda1d\" data-element_type=\"widget\" data-e-type=\"widget\" data-settings=\"{&quot;_animation&quot;:&quot;fadeInUp&quot;}\" data-widget_type=\"text-editor.default\">\n\t\t\t\t<div class=\"elementor-widget-container\">\n\t\t\t\t\t\t\t\t\t<h3>Referencias:<\/h3>\n<ol>\n<li>Giugliani R. RGX-121 gene therapy for the treatment of severe mucopolysaccharidisos type II: CAMPSIITE\u2122 Phase I\/II\/III: A clinical study. Presented at 2022 annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM), Freiburg, Germany, August 31, 2022. Abstract SSIEM22-2719. <\/li>\n<li>Giugliani R. RGX-121 gene therapy for the treatment of severe mucopolysaccharidosis type II: interim analysis of the first in human study. Presented at WORLDSymposium 2022, 18th Annual Research Meeting, San Diego, CA, February 9, 2022.<\/li>\n<li>Escolar M, Phillips D, Cho Y, Mulatya C, Nevoret M-L, Poe M. Natural history of neurodevelopment in neuronopathic mucopolysaccharidosis type II (MPS II): Mullen Scales of Early Learning (MSEL) visual reception, expressive and receptive language and fine motor scale developmental trajectories. Presented at 2022 annual symposium of the Society for the Study of Inborn Errors of Metabolism (SSIEM), Freiburg, Germany, August 2022. Abstract SSIEM22-2814. <\/li>\n<\/ol>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t<\/section>\n\t\t\t\t<\/div>\n\t\t","protected":false},"excerpt":{"rendered":"<p>CAMPSIITE\u2122 es un estudio multic\u00e9ntrico, abierto, de un solo grupo, de fase I\/II\/III en el que se inscribi\u00f3 a ni\u00f1os varones con MPS II neuronop\u00e1tica, de 4 meses de edad hasta los 5 a\u00f1os de edad, para evaluar la eficacia, seguridad y farmacodin\u00e1mica (FD) de la terapia g\u00e9nica RGX-121. Resumen de los puntos clave Los [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_genesis_hide_title":false,"_genesis_hide_breadcrumbs":false,"_genesis_hide_singular_image":false,"_genesis_hide_footer_widgets":false,"_genesis_custom_body_class":"","_genesis_custom_post_class":"","_genesis_layout":"","footnotes":""},"class_list":{"0":"post-440","1":"page","2":"type-page","3":"status-publish","5":"entry"},"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.5 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Campsiite: Phase I\/II\/III Study of RGX-121 for MPS II (Hunter Syndrome)<\/title>\n<meta name=\"description\" content=\"Healthcare Providers - CAMPSIITE\u2122 is a Phase I\/II\/III study enrolling boys with neuronopathic MPS II, to evaluate the efficacy, safety, and PD of RGX-121 gene therapy.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/campsiitestudy.com\/es\/para-profesionales-sanitarios\/\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Campsiite: Phase I\/II\/III Study of RGX-121 for MPS II (Hunter Syndrome)\" \/>\n<meta property=\"og:description\" content=\"Healthcare Providers - 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