{"id":4142,"date":"2025-07-17T12:11:19","date_gmt":"2025-07-17T09:11:19","guid":{"rendered":"https:\/\/dmdwarrior.com\/?p=4142"},"modified":"2025-11-03T20:21:36","modified_gmt":"2025-11-03T17:21:36","slug":"insmed-ins1201-gene-therapy-phase-1-clinical-trial-nct06817382","status":"publish","type":"post","link":"https:\/\/dmdwarrior.com\/pt\/insmed-ins1201-gene-therapy-phase-1-clinical-trial-nct06817382\/","title":{"rendered":"In\u00edcio do ensaio cl\u00ednico de fase 1 da terapia gen\u00e9tica INS1201"},"content":{"rendered":"<p class=\"wp-block-paragraph\">A Insmed anunciou na p\u00e1gina publicada em ClinicalTrials.gov com o ID NCT06817382 que os ensaios de fase 1 da terapia gen\u00e9tica INS1201 desenvolvida para Distrofia Muscular de Duchenne ser\u00e3o iniciados.<\/p>\n\n\n\n<div class=\"wp-block-rank-math-toc-block\" id=\"rank-math-toc\"><h2>\u00cdndice<\/h2><nav><ul><li><a href=\"#bbm-d-101-gene-therapy-phase-1-2-clinical-trial\">Ensaio cl\u00ednico de fase 1 da terapia gen\u00e9tica INS1201<\/a><\/li><li><a href=\"#what-is-insmed-ins-1201-gene-therapy\">O que \u00e9 a terapia gen\u00e9tica Insmed INS1201?<\/a><\/li><li><a href=\"#bbm-d-101-clinical-trial-nct-07058662\">Ensaio Cl\u00ednico INS1201 \u2013 NCT06817382<\/a><\/li><li><a href=\"#bbm-d-101-phase-1-2-participation-criteria\">Crit\u00e9rios de participa\u00e7\u00e3o da Fase 1 do INS1201<\/a><\/li><li><a href=\"#how-can-i-contact-with-belief-bio-med\">Como posso entrar em contato com a Insmed?<\/a><\/li><\/ul><\/nav><\/div>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"bbm-d-101-gene-therapy-phase-1-2-clinical-trial\">Ensaio cl\u00ednico de fase 1 da terapia gen\u00e9tica INS1201<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">O objetivo principal deste estudo \u00e9 avaliar a seguran\u00e7a e a tolerabilidade de uma dose \u00fanica de INS1201 via administra\u00e7\u00e3o IT em participantes ambulatoriais do sexo masculino com DMD.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"what-is-insmed-ins-1201-gene-therapy\">O que \u00e9 a terapia gen\u00e9tica Insmed INS1201?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">O INS1201 tem como alvo o sistema nervoso central diretamente e \u00e9 administrado intratecalmente, em contraste com o Elevidys, que precisa de inje\u00e7\u00e3o intravenosa sist\u00eamica. Os problemas de seguran\u00e7a e efic\u00e1cia com tratamentos gen\u00e9ticos sist\u00eamicos devem ser abordados por esta estrat\u00e9gia de entrega focada.<\/p>\n\n\n\n<p class=\"wp-block-paragraph\">A administra\u00e7\u00e3o intratecal reduz drasticamente a dose necess\u00e1ria em dez a cinquenta vezes, prevenindo toxicidade sist\u00eamica e efeitos fora do alvo ao concentrar a medica\u00e7\u00e3o onde ela \u00e9 necess\u00e1ria. Apesar de obst\u00e1culos como les\u00e3o da medula espinhal, dano nervoso e risco de infec\u00e7\u00f5es, isso pode fornecer aos pacientes uma op\u00e7\u00e3o mais segura e possivelmente mais bem-sucedida, especialmente \u00e0 luz de quest\u00f5es de seguran\u00e7a associadas \u00e0s grandes dosagens de vetores virais utilizadas na terapia intravenosa.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"bbm-d-101-clinical-trial-nct-07058662\">Ensaio Cl\u00ednico INS1201 \u2013 NCT06817382<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>In\u00edcio do estudo (estimado)<\/strong>: 2025-05-31<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Conclus\u00e3o prim\u00e1ria (estimada)<\/strong>: 2028-01-31<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Conclus\u00e3o do estudo (estimada)<\/strong>: 2028-03-31<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>N\u00famero de participantes (estimado)<\/strong>: 12 (Masculino)<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Locais<\/strong>: EUA<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Idades eleg\u00edveis para estudo<\/strong>: 2 anos a 4 anos (crian\u00e7a)<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"bbm-d-101-phase-1-2-participation-criteria\">Crit\u00e9rios de participa\u00e7\u00e3o da Fase 1 do INS1201<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\">As pessoas devem cumprir os requisitos de inclus\u00e3o para serem eleg\u00edveis. Se uma pessoa tiver outra doen\u00e7a ou estiver em tratamento que a impe\u00e7a de receber terapia gen\u00e9tica, ela poder\u00e1 n\u00e3o participar. O conjunto completo de crit\u00e9rios de inclus\u00e3o e exclus\u00e3o pode ser encontrado clicando no link deste estudo \u2013 <a href=\"https:\/\/clinicaltrials.gov\/study\/NCT06817382\" target=\"_blank\" rel=\"noreferrer noopener\">NCT06817382<\/a> \u2013.<\/p>\n\n\n\n<h2 class=\"wp-block-heading\" id=\"how-can-i-contact-with-belief-bio-med\">Como posso entrar em contato com a Insmed?<\/h2>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Nome<\/strong>:Informa\u00e7\u00f5es m\u00e9dicas do Insmed<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>N\u00famero de telefone<\/strong>: 18444467633<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>E-mail<\/strong>: medicalinformation@insmed.com<\/p>\n\n\n\n<p class=\"wp-block-paragraph\"><strong>Ler mais<\/strong>:\u00a0<a href=\"https:\/\/dmdwarrior.com\/pt\/dmd-therapies\/\" target=\"_blank\" rel=\"noreferrer noopener\">Ensaios cl\u00ednicos para distrofia muscular de Duchenne (lista de todas as pesquisas)<\/a><\/p>","protected":false},"excerpt":{"rendered":"<p>Insmed was announced on the page published on ClinicalTrials.gov with the ID NCT06817382 that the phase 1 trials of INS1201 gene therapy developed for Duchenne Muscular Dystrophy will be initiated. INS1201 Gene Therapy Phase 1 Clinical Trial The primary objective of this study is to evaluate the safety and tolerability of a single dose of [&hellip;]<\/p>\n","protected":false},"author":1,"featured_media":4148,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[27],"tags":[137,61,173,174,172],"class_list":["post-4142","post","type-post","status-publish","format-standard","has-post-thumbnail","category-research","tag-clinical-trial","tag-gene-therapy","tag-ins1201","tag-ins1201-gene-therapy","tag-insmed"],"_links":{"self":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4142","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/comments?post=4142"}],"version-history":[{"count":0,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/posts\/4142\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media\/4148"}],"wp:attachment":[{"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/media?parent=4142"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/categories?post=4142"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/dmdwarrior.com\/pt\/wp-json\/wp\/v2\/tags?post=4142"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}