{"id":5479,"date":"2025-10-13T10:20:41","date_gmt":"2025-10-13T10:20:41","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/?page_id=5479"},"modified":"2026-06-08T14:29:52","modified_gmt":"2026-06-08T14:29:52","slug":"neuronal-ceroid-lipofuscinosis-type-2-cln2-clinical-program","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/neuronal-ceroid-lipofuscinosis-type-2-cln2\/neuronal-ceroid-lipofuscinosis-type-2-cln2-clinical-program\/","title":{"rendered":"Neuronal Ceroid Lipofuscinosis type 2 (CLN2) Clinical Program"},"content":{"rendered":"<div id=\"acf-block-68710d6459d61\" class=\"hero hero-no-overlay hero-large-first-para\">\n            <div class=\"hero-background-image hero-background-image-desktop\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/Program-banner-DNA.png?v=1.27);\"><\/div>\n\t    <div class=\"hero-background-image hero-background-image-mobile\" style=\"background-image: url(https:\/\/medical.biomarin.com\/en-us\/wp-content\/uploads\/sites\/2\/2025\/10\/Program-banner-DNA-Mobile.png?v=1.27);\"><\/div>\n    \t<div class=\"overlay\"><\/div>\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"hero-content\">\n\t\t\t    \t\t\t        <ul class=\"breadcrumb\">\n\t\t\t            \t\t\t                                                                        <li><a href=\"https:\/\/medical.biomarin.com\/en-us\/neuronal-ceroid-lipofuscinosis-type-2-cln2\/\">Neuronal Ceroid Lipofuscinosis type 2 (CLN2)<\/a><\/li>\n                            \t\t\t                                                                        <li>Neuronal Ceroid Lipofuscinosis type 2 (CLN2) Clinical Program<\/li>\n                            \t\t\t            \t\t\t        <\/ul>\n\t\t\t    \t\t\t\t\t\t\t\t\t\t\t\t    <h1>Clinical Program\n<\/h1>\n\t\t\t\t\t\t\t\t\t\t\t\t    <p>Learn about CLN2 clinical trials and research at BioMarin<\/p>\n\t\t\t\t\t\t\t\t\n                <p class=\"inline-buttons\">\n                                                        <\/p>\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n\n<div id=\"acf-block-68cc1503e2b9e\" class=\"vblock pipeline pipeline-listing-wrapper\">\n\t<!-- Pipeline Filter Header\n\tIntegration notes:\n\t- Toggle button-filter-active class to filter button when active\n\t- Component here is generic filter header and is also used for resources (filters themselves differ)\n\t-->\n\t<div class=\"block filter-header\">\n\t\t<div class=\"wrapper\">\n\t\t\t<div class=\"inner-wrapper\">\n\t\t\t\t<h1>CLN2 Pipeline<\/h1>\n\t\t\t\t\n\t\t\t\t\t\t\t\t\t<div class=\"filters\">\n\t\t\t\t\t\t<a class=\"button button-filter\" href=\"#\"><span>Search\/Filter<\/span><\/a>\n\t\t\t\t\t\t<div class=\"filters-content\">\n\t\t\t\t\t\t\t<div class=\"wrapper\">\n\t\t\t\t\t\t\t\t<div class=\"inner-wrapper\">\n\t\t\t\t\t\t\t\t\t<form>\n\t\t\t\t\t\t\t\t\t\t<fieldset>\n\t\t\t\t\t\t\t\t\t\t\t<legend>Filter by phase:<\/legend>\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"filter-checkboxes\">\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-phase-observational\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-phase-observational\">Observational<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-phase-1\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-phase-1\">Phase 1<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-phase-2\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-phase-2\">Phase 2<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-phase-3\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-phase-3\">Phase 3<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-phase-4\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-phase-4\">Phase 4<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/fieldset>\n\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<fieldset>\n\t\t\t\t\t\t\t\t\t\t\t\t<legend>Filter by status:<\/legend>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"filter-checkboxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-rec-stat-active-not-recruiting\" value=\"filter-rec-stat-active-not-recruiting\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-rec-stat-active-not-recruiting\">Active, Not Recruiting<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-rec-stat-recruiting\" value=\"filter-rec-stat-recruiting\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-rec-stat-recruiting\">Recruiting<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-rec-stat-enrolling-by-invitation\" value=\"filter-rec-stat-enrolling-by-invitation\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-rec-stat-enrolling-by-invitation\">Enrolling by Invitation<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-rec-stat-not-started\" value=\"filter-rec-stat-not-started\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-rec-stat-not-started\">Not started<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"form-item check-item\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"checkbox\" id=\"filter-rec-stat-not-yet-recruiting\" value=\"filter-rec-stat-not-yet-recruiting\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<label for=\"filter-rec-stat-not-yet-recruiting\">Not yet recruiting<\/label>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/fieldset>\n\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t<div class=\"filter-footer\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"filter-footer-reset\">\n\t\t\t\t\t\t\t\t\t\t\t\t<a class=\"button button-text\" href=\"#\">Reset all filters<\/a>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"filter-footer-submit\">\n\t\t\t\t\t\t\t\t\t\t\t\t<a class=\"button button-text\" href=\"#\">Cancel<\/a>\n\t\t\t\t\t\t\t\t\t\t\t\t<input type=\"submit\" class=\"button\" value=\"Apply filters\">\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/form>\n\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t<\/div>\n\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n\n\t<!-- Research Pipeline block -->\n\t<div class=\"block bg-band pipeline-listing-main\">\n\t\t<div class=\"wrapper\">\n\t\t\t<div class=\"inner-wrapper\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t                    \n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-listing pipeline-enzyme-therapies\">\n\t\t\t\t\t\t<h2 class=\"h3\">Enzyme Therapies<\/h2>\n\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5 trial-phase trial-phase\">Trial phase<\/h3>\n\n\t\t\t\t\t\t\t<div class=\"pipeline-group\" data-trial-phase-class=\"trial-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">Program (Trial)<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">Condition<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">Phase<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-cln2-disease filter-tech-observational filter-rec-stat-recruiting observational\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Cerliponase alfa<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(190-504)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>CLN2 Disease<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Observational<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Cerliponase Alfa Observational Study<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>To evaluate long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nCLN2, late infantile neuronal ceroid lipofuscinosis type 2;<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/catalogues.ema.europa.eu\/node\/1605\/administrative-details\" target=\"_blank\">For reference and comprehensive trial information, visit EMA.Europa.eu EUPAS29031<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t<article class=\"pipeline-trial filter-condition-cln2-disease filter-tech-observational filter-rec-stat-active-not-recruiting observational\">\n\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-summary\">\n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-headers-summary\">\n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-product\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<h3 class=\"h5\">Cerliponase alfa<\/h3>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>(190-501)<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-condition\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Condition<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>CLN2 Disease<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-phase\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4 class=\"h5\">Phase<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<li>Observational<\/li>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-details\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Title<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>Cerliponase Alfa Observational Study in the US<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Study Description<\/h4>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p>This is a multicenter, observational study for patients with a confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who intend to be or are currently being treated with cerliponase alfa.<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"detail-boxes\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Primary Outcome Measures*<\/h4>\n<ul>\n<li>To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis type 2<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Diagnosed with CLN2 disease<\/li>\n<li>Currently receiving or plan to begin treatment with cerliponase alfa<\/li>\n<li>Parent\/guardian provides informed written consent if required<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Hypersensitivity<\/li>\n<li>Impact of severe adverse events on motor and language functions<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Currently receiving treatment in another investigational device or drug study<\/li>\n<\/ul>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\n\t\t\t\t\t\t\t\t\t\t\t\t\t<p>* Additional measures\/criteria may apply.<br>\nCLN2, late infantile neuronal ceroid lipofuscinosis type 2; TPP1, tripeptidyl peptidase 1<\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT04476862\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT04476862<\/a><\/p>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>                                            \n\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-action\">                                                \n\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"pipeline-expand\">\n\t\t\t\t\t\t\t\t\t\t\t\t\t<div class=\"trigger\"><\/div>\n\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t<\/article>\n\t\t\t\t\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t\t\t\t\t<\/div>\n\t\t\t\t\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":4712,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-5479","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Neuronal Ceroid Lipofuscinosis type 2 (CLN2) Clinical Program | BioMarin Medical<\/title>\n<meta name=\"description\" content=\"Learn about Neuronal Ceroid Lipofuscinosis type 2 (CLN2) clinical trials and research at BioMarin\" \/>\n<meta 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