{"id":7867,"date":"2023-06-22T10:07:38","date_gmt":"2023-06-22T08:07:38","guid":{"rendered":"https:\/\/www.icta.fr\/cas-clients\/prevention-treatment-of-a-non-serious-dermatological-condition-on-the-foot-2\/"},"modified":"2026-01-14T10:19:02","modified_gmt":"2026-01-14T09:19:02","slug":"a-phase-1-2-open-label-safety-tolerability-and-preliminary-efficacy-study-of-implantation-into-one-eye-of-an-atmp-in-patients-with-retinitis-pigmentosa-due-to-monogenic-mutation","status":"publish","type":"casclients","link":"https:\/\/www.icta.fr\/fr\/cas-clients\/a-phase-1-2-open-label-safety-tolerability-and-preliminary-efficacy-study-of-implantation-into-one-eye-of-an-atmp-in-patients-with-retinitis-pigmentosa-due-to-monogenic-mutation\/","title":{"rendered":"A phase 1\/2, open-label, safety, tolerability and preliminary efficacy study of implantation into one eye of an ATMP in patients with retinitis pigmentosa due to monogenic mutation"},"content":{"rendered":"\n
<\/div>
\n

OBJECTIVES<\/h2>\n\n\n\n
\n
\n

PRIMARY OBJECTIVES<\/h3>\n\n\n\n

<\/p>\n\n\n\n

    \n
  • Assess safety and tolerability of the ATMP for 56 weeks after implantation<\/li>\n\n\n\n
  • Curative study: Instant pain relief<\/li>\n<\/ul>\n\n\n\n

    <\/p>\n\n\n\n

      \n
    • <\/li>\n<\/ul>\n\n\n\n

      OBSERVATORY OBJECTIVES<\/h3>\n\n\n\n

      <\/p>\n\n\n\n

        \n
      • Evaluation of photoreceptor survival<\/li>\n\n\n\n
      • Change in visual function (by diagnosys-Full-field stimulus, D-FST)<\/li>\n<\/ul>\n<\/div>\n\n\n\n
        \n

        SECONDARY OBJECTIVES<\/h3>\n\n\n\n

        <\/p>\n\n\n\n

          \n
        • Evaluate placement and position of the patch\n
            \n
          • <\/li>\n<\/ul>\n<\/li>\n\n\n\n
          • Assess preliminary efficacy based on :\n
              \n
            • Evaluation of visual function<\/li>\n\n\n\n
            • Eye fundus<\/li>\n\n\n\n
            • Evaluation of photoreceptor survival<\/li>\n<\/ul>\n<\/li>\n\n\n\n
            • Assess long-term safety, tolerability and efficacy up to 5 years after ATMP implantation<\/li>\n<\/ul>\n<\/div>\n<\/div>\n\n\n\n
              \n
              \n

              SCIENTIFIC and DATA VALORISATION<\/h3>\n\n\n\n
                \n
              • Scientific article (ongoing)<\/li>\n\n\n\n
              • Intermediary analysis (ongoing)<\/li>\n<\/ul>\n<\/div>\n\n\n\n
                \n

                Key factors <\/strong>of success<\/strong><\/h3>\n\n\n\n
                  \n
                • ICTA\u2019s rigorousness<\/li>\n\n\n\n
                • ICTA\u2019s capability to propose innovative solutions for unusual projects\n
                    \n
                  • <\/li>\n<\/ul>\n<\/li>\n<\/ul>\n<\/div>\n<\/div>\n\n\n\n
                    \n
                    \n

                    LESSONS LEARNED to speed up the start-up phase of the next study<\/h3>\n\n\n\n
                      \n
                    • Importance of close collaboration between ICTA and Sponsor and active involvement of Sponsor during the course of the study<\/li>\n\n\n\n
                    • Optimal project coordination: Safety management, logistics, sub-contractors management.<\/li>\n<\/ul>\n<\/div>\n<\/div>\n\n\n\n

                      CHALLENGES<\/h2>\n\n\n\n
                      KEY CHALLENGES<\/th>ICTA’s SOLUTIONS<\/th>ICTA’s ACHIEVEMENTS<\/th><\/tr><\/thead>
                      Recruitment \/ number of center<\/strong>

                      Rare disease and low patient potential.
                       
                      Only one surgeon trained to the procedure in France.<\/td>                      		Communication to patient association<\/strong>
                       
                      Communication with other ophthalmology centers to reach patients.<\/td>                      		7 out of 12 planned patients were implanted.<\/td><\/tr>
                      AE\/SAE notification<\/strong>

                      Timelines for SUSARs notification, all AEs related to ATMP are considered as SUSARs.<\/td>                      		Very close contact with the sites
                      Monitoring visit after the treatment of each patient.<\/td>                      		1 audit performed by an independent third party => 0 Critical finding and 0 observation on safety.<\/td><\/tr>
                      ATMP handling<\/strong>

                      ATMP production was 30 days with a 48-hour stability.<\/td>                      		Strict schedule and communication plan were validated and implemented early in the study.
                       
                      Regular reminders to the site and the laboratory.<\/td>                      		All patients were implanted at the scheduled date.<\/td><\/tr>
                      <\/td><\/td><\/td><\/tr><\/tbody><\/table><\/figure>\n<\/div><\/section>\n\n\n\n
                        \n
                      • <\/li>\n<\/ul>\n","protected":false},"excerpt":{"rendered":"","protected":false},"author":15,"featured_media":10779,"template":"","meta":{"_acf_changed":false,"_editorskit_title_hidden":false,"_editorskit_reading_time":0,"_editorskit_is_block_options_detached":false,"_editorskit_block_options_position":"{}","inline_featured_image":false,"_uag_custom_page_level_css":"","kk_blocks_editor_width":"","_kiokenblocks_attr":"","_kiokenblocks_dimensions":"","footnotes":""},"categories":[],"class_list":["post-7867","casclients","type-casclients","status-publish","has-post-thumbnail","hentry"],"acf":[],"uagb_featured_image_src":{"full":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa.jpg",700,350,false],"thumbnail":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa-128x88.jpg",128,88,true],"medium":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa-300x150.jpg",300,150,true],"medium_large":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa.jpg",700,350,false],"large":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa.jpg",700,350,false],"1536x1536":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa.jpg",700,350,false],"2048x2048":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa.jpg",700,350,false],"post-thumbnail":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa.jpg",700,350,false],"wpm_taille_1":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa-364x257.jpg",364,257,true],"wpm_taille_slider":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa-240x145.jpg",240,145,true],"gform-image-choice-sm":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa.jpg",300,150,false],"gform-image-choice-md":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa.jpg",400,200,false],"gform-image-choice-lg":["https:\/\/www.icta.fr\/wp-content\/uploads\/Retinitis-Pigmentosa.jpg",600,300,false]},"uagb_author_info":{"display_name":"Manon ROBERT","author_link":"https:\/\/www.icta.fr\/fr\/author\/mrobert-icta21\/"},"uagb_comment_info":0,"uagb_excerpt":null,"_links":{"self":[{"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/casclients\/7867","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/casclients"}],"about":[{"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/types\/casclients"}],"author":[{"embeddable":true,"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/users\/15"}],"version-history":[{"count":12,"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/casclients\/7867\/revisions"}],"predecessor-version":[{"id":8970,"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/casclients\/7867\/revisions\/8970"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/media\/10779"}],"wp:attachment":[{"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/media?parent=7867"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.icta.fr\/fr\/wp-json\/wp\/v2\/categories?post=7867"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}