TRIAL OF VALOR RELEASE DATE TRIAL

The gene therapy is a short DNA molecule called an antisense oligonucleotide (ASO) which is capable of selectively binding to and degrading toxic products made from the C9orf72 mutation. The main purpose of this study is to find out about the safety, tolerability, and pharmacokinetics (how the study drug is processed by the body) of the study drug when given as repeated doses in adult patients with C9orf72-associated ALS. This is double blind, multicenter clinical research study is the first time the study drug, BIIB078, will be tested in humans.

TRIAL OF VALOR RELEASE DATE TRIAL

The ATLAS trial will enroll about 150 adults with SOD1 mutations that are typically associated with rapid disease progression. This refers to when a person displays early signs of MND, like nervous system damage, but does not have overt clinical signs of the disease. However, Biogen may need to revise or discontinue this programme if no clear path forward for tofersen is established or if another trial is required.ДЄ Phase 3В clinical trial, called ATLAS, will seek to determine the value and optimal timing to begin treatment with tofersen in people who have SOD1 gene mutations but have not yet started to show symptoms (pre-symptomatic carriers). This means more people with SOD1-MND will be able to access the treatment before completion of the standard clinical trials procedure. October 2021: Biogen announced expansion of the eligibility for its ongoing early access programme to the broader SOD1-MND community. Tofersen: antisense oligonucleotide drug shows promising results in Phase 1/2 trial Emerging Gene-Targeting Therapies for SOD1-ALS

Read more about these trials on our MND Research Blog: The open label extension studyВ of BIIB067 was enrolling by invitation only, now active but not recruiting - find out more on (NCT03070119). Additionally, the results of VALOR and the ongoing open label extension study showed that early access to tofersen led to less decline across secondary outcome measures.ДЇind out more about this trial at (NCT02623699). However, signs of reduced disease progression were seen across multiple secondary and exploratory endpoints, including respiratory function and reduced levels of SOD1 in the CSF. In a press release published on 17 October 2021 Biogen announced their phase 3 trial for tofersen (VALOR) did not meet its primary endpoint. The primary outcomes measured the change in the ALSFRS-R from baseline to week 28. The Phase 3 VALORВ study explored the effects in a greater number of participants in the UK (Sheffield and London) and internationally. The Phase 1/2 studyВ results demonstrated proof-of-concept and proof-of-biology of tofersen. Early analyses showed that delivering the highest dose led to significant reduction of SOD1 protein levels in the cerebrospinal fluid (CSF) and showed signs of slower clinical progression. To date, the drug has shown acceptable safety and tolerability. The first phase of the trial began in early 2016, recruiting 70 people with MND in the USA, Canada and Europe (including one centre in the UK). It uses an approach known as вЂantisenseвЂ™, in which the drug directly interferes with the faulty instructions for making SOD1 protein, thus stopping the production of the disease-causing substance. Phase 3 ATLAS вЂ“ ACTIVE, NOT YET RECRUITING IN UKД«IIB067В (also known as Tofersen), developed by Biogen in collaboration with Ionis Pharmaceuticals, is specifically designed to treat familial MND caused by mistakes in the SOD1 gene. Annual Stephen Hawking Foundation MND Lecture.Dysphagia, nutrition and saliva problems.All-Party Parliamentary Group (APPG) on MND.

MND Matters: the MND Association podcast.

Get people with MND on the extremely vulnerable list.

Useful organisations and internet sites.Information in other languages and formats.Information for people with or affected by MND.How can I check if my treatment and care are appropriate?.Needs assessment, home care and home adaptations.Benefits advice service for motor neurone disease.Guidance for those identified as extremely vulnerable.Covid-19 immunisation and booster jabs for people with MND.

Trial of valor release date