{"id":2086,"date":"2018-09-20T00:36:57","date_gmt":"2018-09-20T00:36:57","guid":{"rendered":"https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/index.php\/2018\/09\/20\/pipeline-ensayos-clinicos\/"},"modified":"2026-03-02T12:02:38","modified_gmt":"2026-03-02T12:02:38","slug":"pipeline-ensayos-clinicos","status":"publish","type":"post","link":"https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/index.php\/2018\/09\/20\/pipeline-ensayos-clinicos\/","title":{"rendered":"Pipeline y evoluci\u00f3n de ensayos cl\u00ednicos"},"content":{"rendered":"<p>&nbsp;<\/p>\n<p style=\"text-align: justify;\">La INVESTIGACI\u00d3N es nuestra esperanza para encontrar una cura o tratamiento, o al menos mejorar la calidad de vida de los pacientes con ataxia telangiectasia.<\/p>\n<p style=\"text-align: justify;\">Un equipo de familias de Aefat, en colaboraci\u00f3n con algunos expertos, ha preparado el siguiente <em><strong>PIPELINE*<\/strong><\/em>: un gr\u00e1fico que muestra el progreso de la investigaci\u00f3n, los ensayos cl\u00ednicos que se est\u00e1n desarrollando en todo el mundo, y los principales tratamientos que se est\u00e1n investigando en relaci\u00f3n con la ataxia telangiectasia. Iremos actualizando este <em>pipeline<\/em> peri\u00f3dicamente para que todas las familias e investigadores tengan acceso a esta informaci\u00f3n.<\/p>\n<p style=\"text-align: justify;\"><img fetchpriority=\"high\" decoding=\"async\" class=\" size-full wp-image-2084\" src=\"https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/pipeline_aefat_ataxia_telangiectasia_English2.jpg\" alt=\"pipeline_aefat_ataxia_telangiectasia_English\" usemap=\"#mapa1\" width=\"1085\" height=\"482\" srcset=\"https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/pipeline_aefat_ataxia_telangiectasia_English2.jpg 1085w, https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/pipeline_aefat_ataxia_telangiectasia_English2-300x133.jpg 300w, https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/pipeline_aefat_ataxia_telangiectasia_English2-1024x455.jpg 1024w, https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/pipeline_aefat_ataxia_telangiectasia_English2-768x341.jpg 768w, https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/pipeline_aefat_ataxia_telangiectasia_English2-18x8.jpg 18w\" sizes=\"(max-width: 1085px) 100vw, 1085px\" \/><\/p>\n<map name=\"mapa1\">\n<area alt=\"N-Acetyl L-Leucine\" coords=\"70,130,405,155\" shape=\"rect\" href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03759678?term=IntraBio\" target=\"_blank\" title=\"N-Acetyl L-Leucine\" \/>\n<area alt=\"Intrabio, UK\" coords=\"550,130,900,155\" shape=\"rect\" href=\"https:\/\/intrabio.com\/431-2\/\" target=\"_blank\" title=\"Intrabio, UK\" \/>\n<area alt=\"Nicotinamide Riboside (NAD+)\" coords=\"70,155,405,180\" shape=\"rect\" href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04870866?cond=Ataxia+Telangiectasia&amp;draw=2&amp;rank=2\" target=\"_blank\" title=\"Nicotinamide Riboside (NAD+)\" \/>\n<area alt=\"Oslo University Hospital, Norway\" coords=\"550,155,900,180\" shape=\"rect\" href=\"https:\/\/www.med.uio.no\/klinmed\/english\/people\/aca\/hildni\/\" target=\"_blank\" title=\"Oslo University Hospital, Norway\" \/>\n<area alt=\"Triheptanoin\" coords=\"70,180,450,205\" shape=\"rect\" href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04513002?cond=Ataxia+Telangiectasia&amp;draw=2&amp;rank=1\" target=\"_blank\" title=\"Triheptanoin\" \/>\n<area alt=\"Queensland Children\u2019s Hospital Brisbane, Australia\" coords=\"550,180,900,205\" shape=\"rect\" href=\"https:\/\/www.researchgate.net\/profile\/David-Coman-2\" target=\"_blank\" title=\"Queensland Children\u2019s Hospital Brisbane, Australia\" \/>\n<area alt=\"Intra-Erythrocyte Dexamethasone\" coords=\"70,205,500,230\" shape=\"rect\" href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03563053?cond=Ataxia+Telangiectasia&amp;draw=2&amp;rank=8\" target=\"_blank\" title=\"Intra-Erythrocyte Dexamethasone\" \/>\n<area alt=\"Erydel, Italy\" coords=\"550,205,900,230\" shape=\"rect\" href=\"https:\/\/www.erydel.com\/home.php\" target=\"_blank\" title=\"Erydel, Italy\" \/>\n<area alt=\"Hematopoietic stem cell transplant &amp; FiCAT\" coords=\"70,230,325,255\" shape=\"rect\" href=\"https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/index.php\/2021\/07\/13\/universitat-pompeu-fabra-clinica-universidad-de-navarra-y-aefat-investigar-ataxia-telangiectasia\/\" target=\"_blank\" title=\"Hematopoietic stem cell transplant &amp; FiCAT\" \/>\n<area alt=\"CUN (Navarra) &amp; UPF (Barcelona), Spain\" coords=\"550,230,900,255\" shape=\"rect\" href=\"https:\/\/integra-tx.com\/\" target=\"_blank\" title=\"CUN (Navarra) &amp; UPF (Barcelona), Spain\" \/>\n<area alt=\"Nanoparticle chemistry with gene augmentation\" coords=\"70,255,300,280\" shape=\"rect\" href=\"https:\/\/actionforat.org\/brain-penetrating-atm-gene-therapy\/\" target=\"_blank\" title=\"Nanoparticle chemistry with gene augmentation\" \/>\n<area alt=\"University of Nottingham, UK\" coords=\"550,255,900,280\" shape=\"rect\" href=\"https:\/\/www.researchgate.net\/profile\/James-Dixon-8\" target=\"_blank\" title=\"University of Nottingham, UK\" \/>\n<area alt=\"Transposons method\" coords=\"70,280,305,305\" shape=\"rect\" href=\"https:\/\/actionforat.org\/gene-therapy-in-at\/\" target=\"_blank\" title=\"Transposons method\" \/>\n<area alt=\"University of Granada, Spain\" coords=\"550,280,900,305\" shape=\"rect\" href=\"https:\/\/www.ugr.es\/personal\/ignacio-jesus-molina-pineda-infantas\" target=\"_blank\" title=\"University of Granada, Spain\" \/>\n<area alt=\"T-Car\" coords=\"70,325,300,330\" shape=\"rect\" href=\"https:\/\/actionforat.org\/immunotherapy-to-treat-cancer\/\" target=\"_blank\" title=\"T-Car\" \/>\n<area alt=\"Sheba Medical Centre, Israel\" coords=\"550,305,900,330\" shape=\"rect\" href=\"https:\/\/www.shebaonline.org\/doctors\/elad-jacoby\/\" target=\"_blank\" title=\"Sheba Medical Centre, Israel\" \/>\n<area alt=\"Splice site mutations\" coords=\"70,330,475,355\" shape=\"rect\" href=\"https:\/\/actionforat.org\/aso-therapy\/\" target=\"_blank\" title=\"Splice site mutations\" \/>\n<area alt=\"Harvard and Boston Children\u2019s Hospital, USA\" coords=\"550,330,900,355\" shape=\"rect\" href=\"https:\/\/www.theyulab.org\/antisense-therapy\" target=\"_blank\" title=\"Harvard and Boston Children\u2019s Hospital, USA\" \/>\n <\/map>\n<\/p>\n<p><a href=\"pipeline.html\" target=\"Blank\">Abrir a mayor tama\u00f1o<\/a><\/p>\n<p style=\"text-align: justify;\">*Accede a los enlaces de la informaci\u00f3n sobre los investigadores, centros o laboratorios en esta versi\u00f3n en pdf:<\/p>\n<p style=\"text-align: justify;\">&nbsp;{phocadownload view=file|id=139|text=Pipeline Ataxia Telangiectasia Aefat English Version LINKS|target=s}<\/p>\n<h4 style=\"text-align: justify;\"><strong><span style=\"color: #ff00ff;\">MAPA DE TRATAMIENTOS<\/span><\/strong><\/h4>\n<p style=\"text-align: justify;\">Este mapa de caracter\u00edsticas de la ataxia telangiectasia y los posibles tratamientos que existen en la actualidad ha sido elaborado tambi\u00e9n por un equipo de familiares dedicados a colaborar en los avances en investigaci\u00f3n, que ha contado con la revisi\u00f3n y el apoyo de especialistas internacionales.<\/p>\n<p><img decoding=\"async\" class=\" size-full wp-image-2085\" src=\"https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/AT-TREATMENT_POSSIBILITIES_ultima_version_17-01-24-scaled.jpg\" alt=\"AT TREATMENT POSSIBILITIES \u00faltima versi\u00f3n 17 01 24\" width=\"5624\" height=\"2812\" style=\"display: block; margin-left: auto; margin-right: auto;\" srcset=\"https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/AT-TREATMENT_POSSIBILITIES_ultima_version_17-01-24-scaled.jpg 2560w, https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/AT-TREATMENT_POSSIBILITIES_ultima_version_17-01-24-300x150.jpg 300w, https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/AT-TREATMENT_POSSIBILITIES_ultima_version_17-01-24-1024x512.jpg 1024w, https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/AT-TREATMENT_POSSIBILITIES_ultima_version_17-01-24-768x384.jpg 768w, https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/AT-TREATMENT_POSSIBILITIES_ultima_version_17-01-24-1536x768.jpg 1536w, https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/AT-TREATMENT_POSSIBILITIES_ultima_version_17-01-24-2048x1024.jpg 2048w, https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/wp-content\/uploads\/2018\/09\/AT-TREATMENT_POSSIBILITIES_ultima_version_17-01-24-18x9.jpg 18w\" sizes=\"(max-width: 5624px) 100vw, 5624px\" \/><\/p>\n<p>&nbsp;<\/p>\n<h4 style=\"text-align: justify;\"><strong><span style=\"color: #ff00ff;\">ENSAYOS CL\u00cdNICOS DESARROLLADOS EN ESPA\u00d1A<\/span><\/strong><\/h4>\n<p>Desde Aefat, facilitamos la participaci\u00f3n de nuestros afectados en los dos \u00fanicos <strong>ensayos cl\u00ednicos internacionales<\/strong> que se est\u00e1n desarrollando en Espa\u00f1a, ambos coordinados por el Dr. Samuel Ignacio Pascual, Jefe del Servicio de Neurolog\u00eda Pedi\u00e1trica del Hospital La Paz de Madri y profesor asociado del Departamento de Pediatr\u00eda de la Universidad Aut\u00f3noma de Madrid:<\/p>\n<ul>\n<li><span style=\"background-color: #ffff00;\"><a href=\"index.php\/a-t\/investigacion\/ensayo-clinico-la-paz\" style=\"background-color: #ffff00;\">EryDel<\/a><\/span> (Italia), desde 2017. Basado en la introducci\u00f3n de corticoesteroides (dexametasona) directamente en los gl\u00f3bulos rojos de los pacientes. Tratamiento pendiente de aprobaci\u00f3n por FDA (EEUU) y EMA (Europa). El estudio realizado en Italia en Fase II demostr\u00f3 que frena el avance degenerativo de la ataxia y la alteraci\u00f3n del movimiento ocular, y el paciente mejora a nivel motriz y en equilibrio. Se dise\u00f1\u00f3 un modo especial de introducirlos directamente en los gl\u00f3bulos rojos (eritrocitos), y los resultados observados en el ensayo terap\u00e9utico italiano en fase II (menos pacientes y sin grupo placebo) y a nivel internacional en Fase III han demostrado que los beneficios se mantienen durante m\u00e1s tiempo y sin los efectos secundarios que supone el tratamiento por v\u00eda oral.<\/li>\n<\/ul>\n<p>&nbsp; &nbsp; &nbsp; &nbsp;Los pacientes que participaron en Espa\u00f1a est\u00e1n continuando en uso expandido hasta que el tratamiento est\u00e9 aprobado y pueda comercializarse.<\/p>\n<p style=\"text-align: justify; padding-left: 30px;\"><strong>ALGUNAS PUBLICACIONES EN MEDIOS DE COMUNICACI\u00d3N:<\/strong><\/p>\n<p style=\"text-align: justify; padding-left: 30px;\"><a href=\"https:\/\/forbes.es\/ultima-hora\/237555\/erydel-presenta-novedades-normativas-sobre-erydex-para-el-tratamiento-de-la-ataxia-telangiectasia\/\">EryDel presenta novedades normativas sobre EryDex para el tratamiento de la ataxia telangiectasia<\/a> (Forbes, 2023 Feb.)<\/p>\n<p style=\"padding-left: 30px;\"><a href=\"index.php?option=com_weblinks&amp;view=weblink&amp;id=118;un-ensayo-clinico-mundial-sobre-ataxia-telangiectasia-inicia-su-fase-iii&amp;catid=25;comunicacion&amp;Itemid=101&amp;task=weblink.go\" target=\"_blank\" rel=\"noopener noreferrer\">Un ensayo cl\u00ednico mundial sobre ataxia telangiectasia inicia su fase III&nbsp;<\/a>(<a href=\"index.php?option=com_weblinks&amp;view=weblink&amp;id=119;diario-medico&amp;catid=26;medios-de-comunicacion&amp;Itemid=101&amp;task=weblink.go\" target=\"_blank\" rel=\"noopener noreferrer\">Diario M\u00e9dico<\/a>, 2017)<\/p>\n<p style=\"padding-left: 30px;\"><a href=\"index.php?option=com_weblinks&amp;view=weblink&amp;id=120;h-la-paz-inicia-un-nuevo-ensayo-medico-para-intentar-frenar-el-avance-degenerativo-de-la-ataxia-telangiectasia&amp;catid=25;comunicacion&amp;Itemid=101&amp;task=weblink.go\" target=\"_blank\" rel=\"noopener noreferrer\" title=\"H. La Paz inicia un nuevo ensayo m\u00e9dico para intentar frenar el avance degenerativo de la ataxia telangiectasia\">H. La Paz inicia un nuevo ensayo m\u00e9dico para intentar frenar el avance degenerativo de la ataxia telangiectasia<\/a>&nbsp;(<a href=\"index.php?option=com_weblinks&amp;view=weblink&amp;id=67;infosalus&amp;catid=26;medios-de-comunicacion&amp;Itemid=101&amp;task=weblink.go\" target=\"_blank\" rel=\"noopener noreferrer\">Infosalus<\/a>, 2017)<\/p>\n<ul>\n<li><span style=\"background-color: #ffff00;\"><a href=\"https:\/\/exciting-elbakyan.137-74-93-36.plesk.page\/index.php\/2020\/12\/14\/intrabio-ensayo-clinico-ataxia-telangiectasia\/\" style=\"background-color: #ffff00;\">Intrabio<\/a><\/span> (Reino Unido), desde 2019. El ensayo cl\u00ednico&nbsp;<strong><a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03759678?cond=acetyl+leucine&amp;draw=2&amp;rank=1\">IB1001-203<\/a><\/strong><strong>\u201cEfectos de la N-acetil-L-leucina (IB1001) en la ataxia telangiectasia\u201d<\/strong>es un ensayo de fase II, abierto, multinacional y multic\u00e9ntrico, enmascarado para el evaluador. Investiga los efectos del nuevo f\u00e1rmaco IB1001 (N-Acetil-L-Leucina) para el tratamiento de la ataxia telangiectasia (AT).<\/li>\n<\/ul>\n<p style=\"padding-left: 30px;\">M\u00e1s informaci\u00f3n en <a href=\"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT03759678?term=IntraBio\">Clinical Trials. gov<\/a><\/p>\n<p style=\"padding-left: 30px;\">M\u00e1s informaci\u00f3n en <a href=\"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/orphan-designations\/eu3182124\">EMA <\/a>(Agencia Europea del Medicamento)&nbsp;<\/p>\n<p style=\"text-align: justify;\"><b>OTROS ENSAYOS CL\u00cdNICOS RELACIONADOS CON LA AT (ACTUALES O TERMINADOS)<\/b><\/p>\n<p style=\"text-align: justify;\"><a href=\"https:\/\/clinicaltrials.gov\/ct2\/results?cond=%22Ataxia+telangiectasia%22\">https:\/\/clinicaltrials.gov\/ct2\/results?cond=%22Ataxia+telangiectasia%22<\/a><\/p>\n<p><strong><span style=\"color: #ff00ff;\">M\u00c1S INFORMACI\u00d3N<\/span> SOBRE PROYECTOS DE INVESTIGACI\u00d3N, ENSAYOS CL\u00cdNICOS Y RECURSOS INTERNACIONALES<\/strong> en la web <a href=\"https:\/\/www.cureat.org\">www.cureat.org<\/a>&nbsp;(A-T Global Alliance de la que Aefat es fundadora y forma parte desde 2020)<\/p>\n<p>\u00ab<\/p>\n","protected":false},"excerpt":{"rendered":"<p>&nbsp; La INVESTIGACI\u00d3N es nuestra esperanza para encontrar una cura o tratamiento, o al menos mejorar la calidad de vida de los pacientes con ataxia telangiectasia. Un equipo de familias de Aefat, en colaboraci\u00f3n con algunos expertos, ha preparado el siguiente PIPELINE*: un gr\u00e1fico que muestra el progreso de la investigaci\u00f3n, los ensayos cl\u00ednicos que [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":2084,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"footnotes":""},"categories":[811],"tags":[278,643,642,116,644,428,645],"class_list":["post-2086","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-investigacion","tag-ataxia-telangientasia","tag-clinical-trial","tag-ensayos-clinicos","tag-investigacion","tag-pipeline","tag-research","tag-tratamientos"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.2 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Pipeline y evoluci\u00f3n de ensayos cl\u00ednicos - Aefat<\/title>\n<meta name=\"robots\" content=\"noindex, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<meta property=\"og:locale\" content=\"es_ES\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Pipeline y evoluci\u00f3n de ensayos cl\u00ednicos - Aefat\" \/>\n<meta property=\"og:description\" content=\"&nbsp; La INVESTIGACI\u00d3N es nuestra esperanza para encontrar una cura o tratamiento, o al menos mejorar la calidad de vida de los pacientes con ataxia telangiectasia. 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