{"id":5364,"date":"2025-10-07T14:10:11","date_gmt":"2025-10-07T14:10:11","guid":{"rendered":"https:\/\/medical.biomarin.com\/en-us\/?page_id=5364"},"modified":"2026-01-07T19:41:28","modified_gmt":"2026-01-07T19:41:28","slug":"hemophilia-a-clinical-program","status":"publish","type":"page","link":"https:\/\/medical.biomarin.com\/en-us\/hemophilia-a\/hemophilia-a-clinical-program\/","title":{"rendered":"Hemophilia A 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-drop.png?v=1.20);\"><\/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-drop-Mobile.png?v=1.20);\"><\/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\/hemophilia-a\/\">Hemophilia A<\/a><\/li>\n                            \t\t\t                                                                        <li>Hemophilia A 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 Hemophilia A 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    <!-- Pipeline Filter Header\n    Integration notes:\n    - Toggle button-filter-active class to filter button when active\n    - Component here is generic filter header and is also used for resources (filters themselves differ)\n    -->\n    <div class=\"block filter-header\">\n        <div class=\"wrapper\">\n            <div class=\"inner-wrapper\">\n                <h1>Hemophilia A Pipeline<\/h1>\n                \n                                    <div class=\"filters\">\n                        <a class=\"button button-filter\" href=\"#\"><span>Search\/Filter<\/span><\/a>\n                        <div class=\"filters-content\">\n                            <div class=\"wrapper\">\n                                <div class=\"inner-wrapper\">\n                                    <form>\n                   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check-item\">\n                                                    <input type=\"checkbox\" id=\"filter-phase-1\">\n                                                    <label for=\"filter-phase-1\">Phase 1<\/label>\n                                                <\/div>\n                                                <div class=\"form-item check-item\">\n                                                    <input type=\"checkbox\" id=\"filter-phase-2\">\n                                                    <label for=\"filter-phase-2\">Phase 2<\/label>\n                                                <\/div>\n                                                <div class=\"form-item check-item\">\n                                                    <input type=\"checkbox\" id=\"filter-phase-3\">\n                                                    <label for=\"filter-phase-3\">Phase 3<\/label>\n                                                <\/div>\n                                        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type=\"checkbox\" id=\"filter-rec-stat-terminated\" value=\"filter-rec-stat-terminated\">\n                                                                <label for=\"filter-rec-stat-terminated\">Terminated<\/label>\n                                                            <\/div>\n                                                                                                                                                                                                                                                                                                                                                <div class=\"form-item check-item\">\n                                                                <input type=\"checkbox\" id=\"filter-rec-stat-completed\" value=\"filter-rec-stat-completed\">\n                                                                <label for=\"filter-rec-stat-completed\">Completed<\/label>\n                                                       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href=\"#\">Reset all filters<\/a>\n                                            <\/div>\n                                            <div class=\"filter-footer-submit\">\n                                                <a class=\"button button-text\" href=\"#\">Cancel<\/a>\n                                                <input type=\"submit\" class=\"button\" value=\"Apply filters\">\n                                            <\/div>\n                                        <\/div>\n                                    <\/form>\n                                <\/div>\n                            <\/div>\n                        <\/div>\n                    <\/div>\n                            <\/div>\n        <\/div>\n    <\/div>\n\n    <!-- Research Pipeline block -->\n    <div class=\"block bg-band pipeline-listing-main\">\n        <div class=\"wrapper\">\n            <div class=\"inner-wrapper\">\n                                                                            \n                                        <div class=\"pipeline-listing pipeline-hemophilia-a\">\n                        <h2 class=\"h3\">Hemophilia A<\/h2>\n\n                                                                                                                                        <h3 class=\"h5 trial-phase trial-phase\">Trial phase<\/h3>\n\n                            <div class=\"pipeline-group\" data-trial-phase-class=\"trial-phase\">\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                                                            <div class=\"pipeline-headers\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">Product (Trial)<\/div>\n                                                <div class=\"pipeline-condition\">Condition<\/div>\n                                                <div class=\"pipeline-technology\">Technology<\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                        <\/div>\n                                    \n                                    \n                                    <article class=\"pipeline-trial pre-ind phase-1 phase-2 filter-condition-hemophilia-a filter-tech-gene-therapy filter-rec-stat-active-not-recruiting\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">Valoctocogene roxaparvovec-rvox<\/h3>\n                                                    <p>(270-205 [GENEr8-INH])<\/p>\n                                                    <p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n                                                <\/div>\n                                                                                                    <div class=\"pipeline-condition\">\n                                                        <h4 class=\"h5\">Condition<\/h4>\n                                                        <p>Hemophilia A<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Gene Therapy<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>This Phase 1\/2 clinical study will evaluate the safety and efficacy of valoctocogene-roxaparvovec-rvox (BMN 270) in patients with severe hemophilia A and inhibitors to FVIII. Part A of the study will include subjects who have active inhibitors to FVIII, and Part B will include subjects with a prior history of inhibitors.<\/p>\n                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Treatment-related adverse events over 60 months<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Males &#x2265;18 years of age with hemophilia A<\/li>\n<li>Documented prior residual FVIII activity &#x2264;1 IU\/dL<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Change in median FVIII activity<\/li>\n<li>Change in FVIII inhibiter titer<\/li>\n<li>Absence of recurrence of FVIII inhibitors<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Detectable pre-existing antibodies to the AAV5 capsid<\/li>\n<li>Active infection or immunosuppressive disorder (patients with HIV and undetectable viral load are not excluded)<\/li>\n<li>Receiving immune tolerance induction therapy or prophylaxis with FVIII (Part A only)<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                    <\/div>\n                                                    \n                                                    <p>* Additional measures\/criteria may apply.<br>\nAAV, adeno-associated virus; FVIII, factor VIII; HIV, human immunodeficiency virus.<\/p>\n                                                                                                            <p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT04684940\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT04684940<\/a><\/p>\n                                                                                                    <\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases pipeline-inline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-expand\">\n                                                    <div class=\"trigger\"><\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/article>\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                    \n                                    \n                                    <article class=\"pipeline-trial pre-ind phase-1 phase-2 phase-3 filter-condition-hemophilia-a filter-tech-gene-therapy filter-rec-stat-active-not-recruiting\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">Valoctocogene roxaparvovec-rvox<\/h3>\n                                                    <p>(270-304 [GENEr8-JPN)<\/p>\n                                                    <p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Active, Not Recruiting<\/span><\/p>\n                                                <\/div>\n                                                                                                    <div class=\"pipeline-condition\">\n                                                        <h4 class=\"h5\">Condition<\/h4>\n                                                        <p>Hemophilia A<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Gene Therapy<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A (GENEr8-JPN)<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>This Phase 3 clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.<\/p>\n                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Change from baseline in FVIII activity<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Japanese males &#x2265;18 years of age with hemophilia A&#x200B;<\/li>\n<li>Residual FVIII levels &#x2264;1 IU\/d&#x200B;L<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Change from baseline in utilization of FVIII replacement therapy&#x200B;<\/li>\n<li>Change from baseline in number of bleeding episodes requiring FVIII replacement treatment&#x200B;<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Detectable pre-existing antibodies to the AAV5 capsid&#x200B;<\/li>\n<li>Active infection or any immunosuppressive disorder, including HIV infection&#x200B;<\/li>\n<li>Significant liver dysfunction&#x200B;<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                    <\/div>\n                                                    \n                                                    <p>* Additional measures\/criteria may apply.<br>\nAAV, adeno-associated virus; FVIII, factor VIII; HIV, human immunodeficiency virus.<\/p>\n                                                                                                            <p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT06224907\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT06224907<\/a><\/p>\n                                                                                                    <\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases pipeline-inline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-expand\">\n                                                    <div class=\"trigger\"><\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/article>\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                    \n                                    \n                                    <article class=\"pipeline-trial pre-ind phase-1 phase-2 phase-3 phase-4 filter-condition-hemophilia-a filter-tech-gene-therapy filter-rec-stat-enrolling-by-invitation\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">Valoctocogene roxaparvovec-rvox<\/h3>\n                                                    <p>(270-401 [GENEr8-LTE])<\/p>\n                                                    <p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Enrolling by Invitation<\/span><\/p>\n                                                <\/div>\n                                                                                                    <div class=\"pipeline-condition\">\n                                                        <h4 class=\"h5\">Condition<\/h4>\n                                                        <p>Hemophilia A<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Gene Therapy<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>The BMN 270 (valoctocogene-roxaparvovec-rvox) clinical development program consists of multiple interventional studies designed to assess the safety and efficacy of a single infusion of valoctocogene-roxaparvovec-rvox for at least 5 years post-infusion. This long-term follow-up study is needed to help further understand the long-term safety of valoctocogene-roxaparvovec-rvox beyond 5 years and to assess the durability of efficacy.<\/p>\n                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Primary Outcome Measure*<\/h4>\n<ul>\n<li>Evaluate the long-term safety of valoctocogene-roxaparvovec-rvox<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Subjects must have completed their primary treatment study<\/li>\n<li>Subjects must be capable of giving signed informed consent<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Secondary Outcome Measures*<\/h4>\n<ul>\n<li>Evaluate the long-term effects of valoctocogene-roxaparvovec-rvox in subjects with hemophilia A previously treated in a BioMarin clinical trial<\/li>\n<li>Evaluate the use of hemostatic agents<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Exclusion Criteria*<\/h4>\n<ul>\n<li>Subjects must enroll in Study 270-401 within 4 months of the date of that study completion visit<\/li>\n<li>Subjects must be healthy and not have a condition that would interfere with interpretation of the study<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                    <\/div>\n                                                    \n                                                    <p>* Additional measures\/criteria may apply.<\/p>\n                                                                                                            <p><a class=\"button button-text button-external\" href=\"https:\/\/clinicaltrials.gov\/study\/NCT05768386\" target=\"_blank\">For reference and comprehensive trial information, visit ClinicalTrials.gov NCT05768386<\/a><\/p>\n                                                                                                    <\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases pipeline-inline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">\n                                                    <div class=\"phase-progress phase-progress-100\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-expand\">\n                                                    <div class=\"trigger\"><\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/article>\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                    \n                                    \n                                    <article class=\"pipeline-trial observational filter-condition-hemophilia-a filter-tech-gene-therapy filter-rec-stat-not-started\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">Valoctocogene roxaparvovec-rvox<\/h3>\n                                                    <p>(270-601 [GENEr8-COAS])<\/p>\n                                                    <p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Not started<\/span><\/p>\n                                                <\/div>\n                                                                                                    <div class=\"pipeline-condition\">\n                                                        <h4 class=\"h5\">Condition<\/h4>\n                                                        <p>Hemophilia A<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Gene Therapy<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>GENEr8-COAS: A Non-Interventional, Multi-National, Longitudinal Study of Patients Treated with Roctavian (valoctocogene roxaparvovec)<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>This study is a non-interventional, multi national, longitudinal cohort study will describe the bleeding profile and long-term durability of coagulation factor VIII (FVIII) expression in subjects administered with Roctavian.<\/p>\n                                                    \n                                                                                                            <div class=\"detail-boxes\">\n                                                                                                                                                                                            <div class=\"box\">\n                                                                    <h4>Select Inclusion Criteria*<\/h4>\n<ul>\n<li>Diagnosed with HA and treated with Roctavian. Patients will be offered the opportunity to enroll in this study only after a clinical decision has been made that they will receive Roctavian, however, only subjects who are eventually administered Roctavian will be included in the analysis.<\/li>\n<\/ul>\n                                                                <\/div>\n                                                                                                                    <\/div>\n                                                    \n                                                    <p>* Additional measures\/criteria may apply.<\/p>\n                                                                                                            <p><a class=\"button button-text button-external\" href=\"https:\/\/catalogues.ema.europa.eu\/node\/3372\/administrative-details\" target=\"_blank\">For reference and comprehensive trial information, visit EMA.Europa.eu EUPAS49071<\/a><\/p>\n                                                                                                    <\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases pipeline-inline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-expand\">\n                                                    <div class=\"trigger\"><\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/article>\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                    \n                                    \n                                    <article class=\"pipeline-trial observational filter-condition-hemophilia-a filter-tech-gene-therapy filter-rec-stat-recruiting\">\n                                        <div class=\"pipeline-summary\">\n                                            <div class=\"pipeline-headers-summary\">\n                                                <div class=\"pipeline-product\">\n                                                    <h3 class=\"h5\">Valoctocogene roxaparvovec-rvox<\/h3>\n                                                    <p>(270-801 [GENEr8-GTR])<\/p>\n                                                    <p class=\"status\"><span class=\"label\">Trial Status<\/span> <span class=\"status\">Recruiting<\/span><\/p>\n                                                <\/div>\n                                                                                                    <div class=\"pipeline-condition\">\n                                                        <h4 class=\"h5\">Condition<\/h4>\n                                                        <p>Hemophilia A<\/p>\n                                                    <\/div>\n                                                                                                                                                    <div class=\"pipeline-technology\">\n                                                        <h4 class=\"h5\">Technology<\/h4>\n                                                        <ul>\n                                                            <li>Gene Therapy<\/li>\n                                                        <\/ul>\n                                                    <\/div>\n                                                                                                <div class=\"pipeline-details\">\n                                                                                                            <h4>Title<\/h4>\n                                                        <p>GENEr8-GTR: A Retrospective Cohort Study of Patients Treated with Roctavian(valoctocogene roxaparvovec): An Analysis of Patient Registries<\/p>\n                                                    \n                                                                                                            <h4>Study Description<\/h4>\n                                                        <p>This study is being undertaken to better characterize the long-term effectiveness and safety outcomes of patients treated with Roctavian in a real-world setting<\/p>\n                                                    \n                                                    \n                                                    <p>* Additional measures\/criteria may apply.<\/p>\n                                                                                                            <p><a class=\"button button-text button-external\" href=\"https:\/\/catalogues.ema.europa.eu\/node\/3388\/administrative-details\" target=\"_blank\">For reference and comprehensive trial information, visit EMA.Europa.eu EUPAS49243<\/a><\/p>\n                                                                                                    <\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases pipeline-inline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">Pre-IND<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">Phase 1<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">Phase 2<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">Phase 3<\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">Phase 4<\/div>\n                                                <div class=\"pipeline-expand\"><\/div>\n                                            <\/div>\n                                            <div class=\"pipeline-phases\">\n                                                <div class=\"pipeline-phase pipeline-phase-pre-ind\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-1\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-2\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-3\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-phase pipeline-phase-4\">\n                                                    <div class=\"phase-progress phase-progress-0\"><div class=\"phase-progress-inner\"><\/div><\/div>\n                                                <\/div>\n                                                <div class=\"pipeline-expand\">\n                                                    <div class=\"trigger\"><\/div>\n                                                <\/div>\n                                            <\/div>\n                                        <\/div>\n                                    <\/article>\n                                                            <\/div>\n                                            <\/div>\n                            <\/div>\n        <\/div>\n    <\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":4701,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-5364","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.1.1 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Hemophilia A Clinical Program | BioMarin Medical<\/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:\/\/medical.biomarin.com\/en-us\/hemophilia-a\/hemophilia-a-clinical-program\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta 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