{"id":426,"date":"2022-08-10T14:19:22","date_gmt":"2022-08-10T18:19:22","guid":{"rendered":"https:\/\/campsiitestudy.com\/prestataires-de-soins-de-sante-uniquement\/"},"modified":"2023-06-08T09:24:12","modified_gmt":"2023-06-08T13:24:12","slug":"prestataires-de-soins-de-sante-uniquement","status":"publish","type":"page","link":"https:\/\/campsiitestudy.com\/fr\/prestataires-de-soins-de-sante-uniquement\/","title":{"rendered":"Prestataires de soins de sant\u00e9 uniquement"},"content":{"rendered":"\t\t<div data-elementor-type=\"wp-page\" data-elementor-id=\"426\" class=\"elementor elementor-426 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-318\" 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-291\" 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-300\" 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-294\" 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\/fr\/\"><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 est une \u00e9tude de phase I\/II\/III multicentrique, ouverte et \u00e0 groupe unique, recrutant des gar\u00e7ons atteints de MPS II neuronopathique, \u00e2g\u00e9s de 4 mois \u00e0 5 ans, visant \u00e0 \u00e9valuer l\u2019efficacit\u00e9, l\u2019innocuit\u00e9 et la pharmacodynamique (PD) de la th\u00e9rapie g\u00e9nique RGX-121.<\/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-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-303\" 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-297\" 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-309\" 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>R\u00e9sum\u00e9 des points cl\u00e9s<\/h3>\n<ul>\n<li>Des donn\u00e9es \u00e9mergentes sur l\u2019histoire naturelle soulignent l\u2019urgence d\u2019un traitement pr\u00e9coce de la MPS II neuronopathique.<\/li>\n<li>Les donn\u00e9es provisoires de la phase I\/II de CAMPSIITE d\u00e9montrent que RGX-121 a \u00e9t\u00e9 bien tol\u00e9r\u00e9 en date du 1er ao\u00fbt 2002. Les donn\u00e9es provisoires concernant la dose s\u00e9lectionn\u00e9e pour l\u2019extension de l\u2019\u00e9tude pivot (Cohorte 3) ont montr\u00e9 des biomarqueurs approchant des taux normaux de GAG<sup>1.<\/sup> <\/li>\n<li>Les donn\u00e9es disponibles des cohortes 1 et 2 ont montr\u00e9 une activit\u00e9 sur le SNC jusqu\u2019\u00e0 2 ans apr\u00e8s l\u2019administration de RGX-121, comme en t\u00e9moignent les am\u00e9liorations de la fonction neurod\u00e9veloppementale et les r\u00e9sultats signal\u00e9s par les soignants. <sup>2<\/sup><\/li>\n<li>CAMPSIITE recrute actuellement des enfants atteints de MPS II neuronopathique \u00e2g\u00e9s de 4 mois \u00e0 5 ans \u00e0 la dose pivot, afin d\u2019\u00e9valuer les taux de GAG dans le liquide c\u00e9phalo-rachidien (LCR) et d\u2019\u00e9valuer l\u2019effet de RGX-121 sur la fonction neurod\u00e9veloppementale.<\/li>\n<li>Si vous avez un patient de moins de 5 ans qui est atteint ou pourrait \u00eatre atteint de MPS II neuronopathique, communiquez avec un centre d\u2019\u00e9tude ou envoyez un courriel \u00e0 <a href=\"mailto:medinfo@regenxbio.com\">medinfo@regenxbio.com<\/a> pour obtenir plus de renseignements.<\/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>Donn\u00e9es sur l\u2019histoire naturelle dans la MPS II<\/h2>\n<p>Une analyse des donn\u00e9es de l\u2019histoire naturelle de 32 patients (\u00e2ge au moment de la premi\u00e8re \u00e9valuation : 1 \u00e0 117 mois) permet de dresser un tableau complet et multidomaines du d\u00e9veloppement de la MPS II neuronopathique.<\/p>\n<p>Les donn\u00e9es d\u00e9montrent que:<\/p>\n<ul>\n<li>L\u2019acquisition des comp\u00e9tences d\u00e9veloppementales ralentit dans toutes les \u00e9chelles d\u2019apprentissage pr\u00e9coce de Mullen (MSEL) par rapport au d\u00e9veloppement des pairs (stade de retard) et l\u2019\u00e9chelle du langage expressif fournit l\u2019indication la plus pr\u00e9coce du retard. <sup>3<\/sup><\/li>\n<li>Les trajectoires des notes d\u2019\u00e2ge (age-equivalent, AEq) moyen de d\u00e9veloppement commencent \u00e0 d\u00e9vier d\u00e8s 12,8 mois. <sup>3<\/sup><\/li>\n<li>Le rythme d\u2019acquisition des comp\u00e9tences ralentit t\u00f4t dans le d\u00e9veloppement et le retard s\u2019accentue avec le temps. <sup>3<\/sup><\/li>\n<\/ul>\n<p>Les donn\u00e9es de l\u2019analyse de l\u2019histoire naturelle ont \u00e9t\u00e9 pr\u00e9sent\u00e9es sous forme d\u2019affiche lors de la r\u00e9union annuelle de 2022 de la Society for the Study of Inborn Errors of Metabolism (SSIEM). <sup>3<\/sup> <a href=\"\/wp-content\/uploads\/2022\/12\/Escolar-MPS-II-Natural-History-n32-SSIEM22-POSTER-vF.pdf\">Cliquez ici pour visualiser l\u2019affiche.<\/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>Occasions d\u2019inscription actuelles<\/h2>\n<p>REGENXBIO recrute des gar\u00e7ons atteints de MPS II neuronopathique, \u00e2g\u00e9s de 4 mois \u00e0 5 ans, pour l\u2019\u00e9tude CAMPSIITE. Les objectifs principaux de cet essai pivot sont d\u2019\u00e9valuer les GAG du liquide c\u00e9phalo-rachidien (LCR) et l\u2019effet de RGX-121 sur la fonction neurod\u00e9veloppementale mesur\u00e9e par les \u00e9chelles de d\u00e9veloppement du nourrisson et de l\u2019enfant en bas \u00e2ge de Bayley, 3e \u00e9dition (Bayley Scales of Infant and Toddler Development, BSID-III), ou par les \u00e9chelles d\u2019apprentissage pr\u00e9coce de Mullen (Mullen Scales of Early Learning, MSEL) chez les enfants atteints de MPS II neuronopathique. L\u2019innocuit\u00e9, les r\u00e9sultats signal\u00e9s par les soignants et les param\u00e8tres d\u2019IRM seront \u00e9galement \u00e9valu\u00e9s.  <\/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>Admissibilit\u00e9<\/h3>\n<p>Les principaux crit\u00e8res d\u2019admissibilit\u00e9 \u00e0 l\u2019essai CAMPSIITE sont indiqu\u00e9s ci-dessous. Pour une liste plus compl\u00e8te des crit\u00e8res d\u2019admissibilit\u00e9, une liste des centres de l\u2019\u00e9tude CAMPSIITE et pour obtenir plus de renseignements, consultez le site <a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\" target=\"_blank\" rel=\"noopener\" data-wplink-edit=\"true\">ClinicalTrials.gov<\/a> (identifiant no 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 Crit\u00e8res principaux d\u2019admissibilit\u00e9\n        <\/th>\n        <td>\n            <p>Les participants admissibles doivent \u00eatre de sexe masculin et \u00e2g\u00e9s de 4 mois \u00e0 5 ans au jour 1 de l\u2019essai, ainsi que pr\u00e9senter un diagnostic document\u00e9 de MPS II neuronopathique. Pour obtenir une liste compl\u00e8te des crit\u00e8res d\u2019admissibilit\u00e9 et des renseignements suppl\u00e9mentaires, veuillez consulter le site <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 le ph\u00e9notype MPS II de votre patient est inconnu, vous pouvez l\u2019orienter vers un centre d\u2019\u00e9tude pour une \u00e9valuation.<\/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 vous avez des patients dans votre cabinet qui pourraient \u00eatre admissibles \u00e0 participer \u00e0 l\u2019\u00e9tude CAMPSIITE, nous vous encourageons &nbsp;\u00e0 communiquer avec un centre d\u2019\u00e9tude.<\/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>Crit\u00e8res d\u2019\u00e9valuation principaux de l\u2019\u00e9tude<\/h3>\n<p>Les participants seront \u00e9valu\u00e9s (innocuit\u00e9 et efficacit\u00e9) pendant deux ans apr\u00e8s le traitement par RGX-121 et seront ensuite encourag\u00e9s \u00e0 participer \u00e0 une \u00e9tude de suivi \u00e0 long terme. Les crit\u00e8res d\u2019\u00e9valuation principaux de l\u2019extension de l\u2019\u00e9tude pivot sont les suivants: <\/p>\n<ul>\n<li>Taux GAG CSF<\/li>\n<li>Fonction neurod\u00e9veloppementale mesur\u00e9e par les \u00e9chelles de d\u00e9veloppement du nourrisson et de l\u2019enfant en bas \u00e2ge de Bayley, 3e \u00e9dition (BSID-III) ou les \u00e9chelles d\u2019apprentissage pr\u00e9coce de Mullen (MSEL)<\/li>\n<\/ul>\n<p>Pour obtenir plus de renseignements au sujet de cet essai, veuillez communiquer par courriel via <a href=\"mailto:medinfo@regenxbio.com\">medinfo@regenxbio.com<\/a> ou consulter le site <a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03566043\" data-wplink-edit=\"true\">ClinicalTrials.gov<\/a> (identifiant no 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>Inscriptions en cours<\/h3><p>Orientez votre patient vers un centre d\u2019\u00e9tude pour qu\u2019il soit \u00e9valu\u00e9.<\/p><p><a class=\"button\" href=\"https:\/\/campsiitestudy.com\/fr\/#campsiite-locations\">Emplacements 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>Nous contacter<\/h3>\n<p>Pour obtenir plus de renseignements sur nos \u00e9tudes pour les patients atteints de MPS II, veuillez communiquer avec nous via l\u2019adresse courriel suivante : <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>R\u00e9f\u00e9rences:<\/h3>\n<ol>\n<li>Giugliani R. RGX-121 gene therapy for the treatment of severe mucopolysaccharidisos type II: Phase I\/II\/III de CAMPSIITE\u2122 : Une \u00e9tude clinique. Pr\u00e9sent\u00e9 lors du symposium annuel 2022 de la Society for the Study of Inborn Errors of Metabolism (SSIEM), Fribourg, Allemagne, 31 ao\u00fbt 2022. R\u00e9sum\u00e9 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. Pr\u00e9sent\u00e9 lors du WORLDSymposium 2022, 18e r\u00e9union de recherche annuelle, San Diego, CA, 9 f\u00e9vrier 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. Pr\u00e9sent\u00e9 lors du symposium annuel 2022 de la Society for the Study of Inborn Errors of Metabolism (SSIEM), Fribourg, Allemagne, ao\u00fbt 2022. R\u00e9sum\u00e9 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 est une \u00e9tude de phase I\/II\/III multicentrique, ouverte et \u00e0 groupe unique, recrutant des gar\u00e7ons atteints de MPS II neuronopathique, \u00e2g\u00e9s de 4 mois \u00e0 5 ans, visant \u00e0 \u00e9valuer l\u2019efficacit\u00e9, l\u2019innocuit\u00e9 et la pharmacodynamique (PD) de la th\u00e9rapie g\u00e9nique RGX-121. R\u00e9sum\u00e9 des points cl\u00e9s Des donn\u00e9es \u00e9mergentes sur l\u2019histoire naturelle soulignent l\u2019urgence d\u2019un [&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-426","1":"page","2":"type-page","3":"status-publish","5":"entry"},"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.6 - 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\/fr\/prestataires-de-soins-de-sante-uniquement\/\" \/>\n<meta property=\"og:locale\" content=\"fr_FR\" \/>\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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