<\/strong>from the website of the International Rett Syndrome Foundation (rettsyndrome.org)<\/p>\nThe new Phase 2 trial will confirm the tolerability and safety of trofinetide in children and will be informative with respect to the planning of a single Phase 3 trial (a pivotal trial) that will include children, adolescents and adults. It will examine higher dose levels than the first Phase 2 trial, the results of which showed trends of increasing clinical benefit with increasing dose levels.<\/p>\n
The primary goal of the study will be to assess the tolerability and safety of the study medication in this younger population. Additionally, Neuren will assess clinical benefit by determining change from baseline on a number of outcome measures including the clinician-completed Motor Behavior Assessment (MBA), the Clinical Global Impression Scale of Improvement and the Caregiver Top 3 Concerns assessment. Each of these measures exhibited clinical benefit in the first study. The trial got underway in early 2016.<\/p>\n
14 November 2016 – Neuren completes enrolment into Phase 2 trial of trofinetide in paediatric Rett syndrome<\/i><\/h4>\n
Neuren Pharmaceuticals announced that enrolment into its Phase 2 clinical trial of trofinetide in paediatric Rett syndrome had been completed. <\/p>\n
The trial is a randomized, double-blind, placebo-controlled Phase 2 clinical trial for girls aged 5 to 15 years with Rett syndrome. It is being conducted at 12 sites in the United States, led by clinicians experienced in the diagnosis and treatment of Rett syndrome. <\/p>\n
Faster enrolment into the trial provided Neuren with the opportunity to expand it beyond the original target of 64 completing subjects, whilst still delivering top-line results in the first quarter of 2017. In total, 82 subjects have been randomized. 62 subjects were randomized into one of four treatment groups: 50mg\/kg, 100mg\/kg, 200mg\/kg and placebo. A further 20 subjects have been randomized into one of two treatment groups: 200mg\/kg and placebo. The total duration of a subject’s participation in the trial, from screening through to follow-up, is eleven weeks. To date, 55 subjects have completed the trial and only one subject has withdrawn before completion.<\/p>\n
30 January 2017 – Neuren completes Phase trial of trofinetide in paediatric Rett syndrome<\/i><\/h4>\n
Neuren Pharmaceuticals announce that the last subject has completed its Phase 2 clinical trial of trofinetide in paediatric Rett syndrome.<\/p>\n
Neuren remains on schedule to receive top-line results from the trial in the second half of March 2017 and soon thereafter intends to engage potential commercial partners regarding the remaining development and commercialisation of trofinetide in the major markets.<\/p>\n
Neuren Executive Chairman Richard Treagus commented “We are grateful for the strong support of the Rett syndrome community, which has helped us to complete the expanded paediatric trial on schedule. We look forward to receiving the results and engaging with clinical experts and potential commercial partners to determine the optimum path to market trofinetide in Rett syndrome.” …………………… <\/p>\n
…………………… Results from two non-clinical toxicity studies of trofinetide will be required prior to initiating extended dosing in a Phase 3 trial and submitting New Drug Applications. The first toxicity study is nearing completion and the second toxicity study is scheduled to commence in mid-2017, concluding in the first half of 2018. Plans are well advanced that would enable manufacturing to commence in the second half of 2017 in preparation for supplying a pivotal Phase 3 trial in Rett syndrome.<\/p>\n","protected":false},"excerpt":{"rendered":"
Today (22 March 2017), Neuren Pharmaceuticals announced to the Australian Stock Exchange in Melbourne that its Phase 2 trial of treatment of Rett syndrome girls aged from 5 to 15 […]<\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_kad_blocks_custom_css":"","_kad_blocks_head_custom_js":"","_kad_blocks_body_custom_js":"","_kad_blocks_footer_custom_js":"","ngg_post_thumbnail":0,"footnotes":""},"categories":[8,12],"tags":[],"taxonomy_info":{"category":[{"value":8,"label":"All News Posts"},{"value":12,"label":"Clinical Trials"}]},"featured_image_src_large":false,"author_info":{"display_name":"Gary Grocott","author_link":"https:\/\/rettaustralia.org.au\/author\/garyg\/"},"comment_info":0,"category_info":[{"term_id":8,"name":"All News Posts","slug":"news","term_group":0,"term_taxonomy_id":8,"taxonomy":"category","description":"","parent":0,"count":40,"filter":"raw","cat_ID":8,"category_count":40,"category_description":"","cat_name":"All News Posts","category_nicename":"news","category_parent":0},{"term_id":12,"name":"Clinical Trials","slug":"clinical-trials","term_group":0,"term_taxonomy_id":12,"taxonomy":"category","description":"","parent":0,"count":6,"filter":"raw","cat_ID":12,"category_count":6,"category_description":"","cat_name":"Clinical Trials","category_nicename":"clinical-trials","category_parent":0}],"tag_info":false,"_links":{"self":[{"href":"https:\/\/rettaustralia.org.au\/wp-json\/wp\/v2\/posts\/940"}],"collection":[{"href":"https:\/\/rettaustralia.org.au\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/rettaustralia.org.au\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/rettaustralia.org.au\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/rettaustralia.org.au\/wp-json\/wp\/v2\/comments?post=940"}],"version-history":[{"count":2,"href":"https:\/\/rettaustralia.org.au\/wp-json\/wp\/v2\/posts\/940\/revisions"}],"predecessor-version":[{"id":5435,"href":"https:\/\/rettaustralia.org.au\/wp-json\/wp\/v2\/posts\/940\/revisions\/5435"}],"wp:attachment":[{"href":"https:\/\/rettaustralia.org.au\/wp-json\/wp\/v2\/media?parent=940"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/rettaustralia.org.au\/wp-json\/wp\/v2\/categories?post=940"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/rettaustralia.org.au\/wp-json\/wp\/v2\/tags?post=940"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}